PsA Patients Have More Medical Visits in Five Years Leading Up to Diagnosis – DocWire News

A new study evaluated healthcare utilization among psoriatic arthritis (PsA) patients in the years leading up to their diagnosis. The researchers concluded here that PsA patients are significantly more likely to have medical visits than other patients during the time period leading up to their diagnosis, going back as far as five years.

PsA is a heterogeneous disease that can present in various clinical manifestations, such as synovitis, enthesitis, dactylitis and spondylitis, the study authors explained. Some of these features can present with only minimal findings on physical examination, and the differentiation from other conditions, such as osteoarthritis, can be challenging. Furthermore, unlike RA and lupus, PsA has no reliable diagnostic biomarkers. These factors, along with the lack of awareness of PsA among patients and primary care physicians, and limited access to specialty care contribute to delays in the diagnosis of PsA.

Better pre-diagnosis may be beneficial to disease treatment and outcomes, the researchers noted, so they explored the burden of musculoskeletal symptoms among patients who were eventually diagnosed with PsA.

The study was a population-based, matched cohort study that collected data from electronic medical records and administrative data in Ontario, Canada. PsA patients were age- and sex-matched to control patients from the same family physicians. Control patients were eligible for inclusion if they did not have a billing code indicating a diagnosis of spondyloarthritis, ankylosing spondylitis, or rheumatoid arthritis. Outcomes included healthcare utilization and costs pertaining to nonspecific musculoskeletal issues over the five-year period leading up to the index date.

Final analysis included 462 PsA patients and 2,310 controls (mean [SD] age, 54.2 [13.8] years; 55.6% were female). PsA patients were significantly more likely to visit a primary care physician for nonspecific musculoskeletal issues in the year leading up to the index date (odds ratio [OR]=2.14; 95% confidence interval [CI], 1.732.64); this trend was also observed in the five years leading up to the index date (OR=1.76; 95% CI, 1.432.18). PsA patients were also more likely than the controls to require musculoskeletal-related specialty care, diagnostic imaging and procedures prior to the index date. PsA patients were more likely to be assessed by nonrheumatologist musculoskeletal specialists (OR range, 1.592.03), visit an emergency department for musculoskeletal-related issues (OR range, 1.332.69), and have joint imaging (OR range, 3.206.26) and joint injections (OR range, 4.639.26).

Over the five years before index date, PsA patients were at least four times more likely than the controls to visit a rheumatologist. The most common diagnosis codes rheumatologists provided were for other disease of the musculoskeletal systems, psoriasis, osteoarthritis, and cramps, leg pain, muscle pain, joint pain, joint swelling.

The study appeared in Arthritis Care & Research.

The study authors concluded of the findings, his pattern reveals some of the underlying causes of diagnosis delays of PsA and highlights the need for diagnostic strategies and novel reliable biomarkers to aid in early diagnosis of PsA.

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PsA Patients Have More Medical Visits in Five Years Leading Up to Diagnosis - DocWire News

Characterization of Patients with Psoriasis in Challenging-to-Treat Body Areas in the Corrona Psoriasis Registry – DocWire News

BACKGROUND:

Real-world studies evaluating patients with challenging-to-treat localizations of psoriasis (scalp, nail, and palmoplantar) are limited.

To characterize patients with versus without psoriasis in challenging-to-treat areas seen in routine US clinical practice.

This retrospective observational study included all adult patients with psoriasis enrolled in the Corrona Psoriasis Registry between April 2015 and May 2018 who initiated a biologic therapy at registry enrollment. Patients were stratified by the presence of scalp, nail, or palmoplantar psoriasis (nonmutually exclusive groups). Patient demographics, clinical char-acteristics, disease activity, and patient-reported outcome measures (pain, fatigue, itch, EuroQol visual analog scale [EQ VAS], Dermatology Life Quality Index [DLQI], and Work Productivity and Activity Impairment questionnaire [WPAI]) were assessed at registry enrollment and compared between patients with versus without each challenging-to-treat area using nonparametric Kruskal-Wallis tests for continuous variables and 2 or Fisher exact tests for categorical variables. Generalized linear regression models were used to estimate differences in disease activity and patient-reported outcomes between patients with versus without each challenging-to-treat area.

Among 2,042 patients with psoriasis (mean age [SD], 49.6 14.7 years; 51.5% male), 38.4% had psoriatic arthritis (PsA), 38.1% had scalp psoriasis, 16.0% had nail psoriasis, 10.9% had palmoplantar psoriasis, and 26.2% had a combination of 2 challenging-to-treat areas and PsA; only 34.2% had body plaque psoriasis without PsA or challenging-to-treat areas. Patients in all challenging-to-treat groups reported higher (mean [95% CI]) itch (scalp, 58.01 [57.62-58.40] vs. 54.35 [53.99-54.72]; nail, 56.42 [56.02-56.81] vs. 55.59 [55.20-55.97]; palmoplantar, 60.22 [59.86-60.59] vs. 55.15 [54.79-55.54]) and lower EQ VAS (scalp, 68.12 [67.78-68.48] vs. 69.46 [69.12-69.81]; nail, 66.21 [65.89-66.55] vs. 69.48 [69.14-69.83]; palmoplantar, 66.21 [66.07-66.75] vs. 69.29 [68.94-69.94]) scores than those without the respective challenging-to-treat localization. Patients with nail or palmoplantar psoriasis reported higher pain, fatigue, and DLQI scores than those without. Higher proportions of patients with scalp or palmoplantar psoriasis reported work impairment compared with those without.

Two-thirds of patients with psoriasis who initiated biologic therapy had PsA and/or 1 challenging-to-treat area. Patients with challenging-to-treat areas had worse patient-reported outcome scores than those without, indicating a significant burden of challenging-to-treat areas on patients quality of life.

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Characterization of Patients with Psoriasis in Challenging-to-Treat Body Areas in the Corrona Psoriasis Registry - DocWire News

Remedies for Psoriasis: THESE home treatments can help you deal with this skin infection – PINKVILLA

Psoriasis is a skin infection that's caused due to multiple reasons. Read below to find out how you can treat psoriasis at home with these doable home remedies.

When it comes to taking care of our skin, we all make sure to apply creams and soaps that maintain the moisture and pH levels of our skin. However, some skin ailments are caused in spite of taking care of our skin. Lifestyle choices, stress and various other factors contribute to these ailments. And one such ailment is psoriasis.

Psoriasis is a skin condition which appears on various parts of the body like knees, elbows, scalp, and the torso. This ailment can be identified when you have thick, red skin with silver-white patches on your body, which are also known as scales. If not taken care of, these patches and scales can get itchy and painful with time. If you are suffering from this ailment, then here are some home remedies for it. However, if the condition worsens it's better to consult a dermatologist.

Here are some home remedies for psoriasis:

When it comes to treating this skin infection at home, plastic wraps can prove to be quite effective. If you are suffering from this skin condition, then you should wrap the affected area with plastic covers, mostly after applying their prescribed medication or ointment. It is done to help the body hold onto the vital natural oils and water.

Sea salt and Epsom salt again helps to deal with psoriasis. Sea salt will ensure removal of thick scales caused due to psoriasis, thus ensuring deeper penetration of the medication into the skin. They are known for their exfoliating properties and hence can provide relief from psoriasis.

Using apple cider vinegar in the affected areas can reduce the itching, pain and burning sensation to a great extent. It is a popular disinfectant and is popular for its properties and considerably benefits people suffering from this condition.

Drinking bitter gourd juice with lime on an empty stomach can again provide some relief from psoriasis. However, one needs to do this daily, since it takes about 5-6 months to show effective results.

You can also treat psoriasis by adding some additional dietary supplements to your routine diet. Vitamin D, aloe vera, fish oil are a few supplements which may help ease psoriasis symptoms.

Reduce fatty snacks and red meat. Add nuts, seeds and foods rich in Omega-3 fatty acids. With an ability to reduce inflammation, theyre the perfect foods for your psoriasis.

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Remedies for Psoriasis: THESE home treatments can help you deal with this skin infection - PINKVILLA

Trader Who Called Bitcoin (BTC) Breakout Says Top Cryptocurrency Poised for Monster Rally to $150,000 – The Daily Hodl

A crypto investor and technical strategist who predicted this months big Bitcoin rally says the king of crypto is targeting a long-term rise to $150,000.

On January 8th, when Bitcoin was trading around $8,000, the anonymous analyst known as Financial Survivalism said BTC appeared to be forming a classic Wycoff Spring pattern. The pattern is named after the late Richard Demille Wyckoff, whose method of analysis relies heavily on laws of supply and demand and divergences between volume and price.

Since then, Bitcoin has rallied to its current price of $9,384, with the analyst now identifying a potential cup-and-handle pattern a formation often used to project a bearish-to-bullish trend reversal.

Bitcoin just confirmed a cup and handle with todays close. If it can reach its target of $11,675 then it would be the first higher high on the weekly in seven months. Breaking down the low of the handle ($8,100) would invalidate the pattern.

The analyst believes BTC is at the outset of a long-term rally that will see the top cryptocurrency match its all-time high by July, and shatter $150,000 by the middle of 2022.

I expect Bitcoin to retest all time highs by July of 2020. It will take some time to breakthrough that level but I fully believe we will before the end of the year. I also a holding onto my longer term target of $150,000 Bitcoin by May 2022.

Meanwhile, analyst Josh Rager says BTC will likely retreat in the short term after moving far and fast this month. But he thinks doom and gloom calls predicting a major trend reversal are starting to get stale.

Pullbacks should be expected, Im fine with shorting, trading the range short term. But these doom and gloom calls get old, clearly Bitcoin broke the downtrend. For now, trading the range is fine.

Featured Image: Shutterstock/Tithi Luadthong

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Trader Who Called Bitcoin (BTC) Breakout Says Top Cryptocurrency Poised for Monster Rally to $150,000 - The Daily Hodl

This Albertan YouTuber Is the Bob Ross of Stealth Camping – VICE

In a YouTube video posted in May 2019, Steve Wallis, a 38 year-old guy who radiates the chuminess of being everyones best friend, offers the helpful tip that, when camping, a good mosquito deterrent is a small fire. He then picks up a half-a-million BTU torch rigged up to a heavy propane tank and ignites a loose assemblage of tree branches and logs, like DiCaprio roasting Nazis with a flamethrower in Once Upon a TimeIn Hollywood. The makeshift bonfire goes up in a flash, cartoonishly orange flames swelling. And that, to nip one of your new favourite YouTubers catchphrases, is camping with Steve.

Like any worthwhile obsession, I came across Wallis while tumbling deep down a YouTube rabbit hole. A few years back, while severely depressed, I mainlined the History Channel reality series Alone, in which survivalists attempt to outlast each other in Canadas most unforgiving hinterlandswith minimal supplies and no crew (all the footage is recorded by the participants with handheld camcorders), armed with their bushwhacking know-how and ability to withstand their own company. (Really, it was this latter facet that kept me hooked: witnessing the ability of the human mind rambling in total isolation.) From there, I developed an interest in camping YouTubers, who similarly recorded their own solo survivalism (mis)adventures. In time, while exploring the niche of people who forgo tents in favour of camping in heavy-duty hammocks, I stumbled into Urban Stealth Camping With Hammock In Residential Area, a video posted by a charming Albertan named Steve Wallis, who calls himself Camping Steve.

These videos were different. The current vogue in survivalismpopularized by programs like Survivorman, Alone and Naked and Afraidvaunt some primal relationship to nature, where the aspiring outdoors-person must start fires from scratch, trap their own food with deadfalls, and fashion sun-shielding bonnets out of woven reeds. Its that romance of (to paraphrase Thoreau) existing more deliberately, of fronting only the essential facts, of sucking all the marrow out of life itself. Its also, in the case of popular programs like Alone or Naked and Afraid, about that more modern romance of going on TV to win a bunch of money.

Camping Steve is no modern primordial man, born naked into natures unfeeling bounty. He camps under tarps in residential areas. He builds rafts out of rain barrels and floats downriver. He hunkers down (another of his favourite turns) in a rented U-Haul in the long-term parking lot of the Edmonton International Airport, braving the elements while avoiding the prying eyes and piercing Maglites of security personnel. He starts fires with hand sanitizer, cooks in closed quarters with propane grills, and is the only camping YouTuber Ive ever seen who rigs his pop-up tent with a CO2 monitor, precisely because he cooks in closed quarters with propane grills. He forgoes both the back-to-the-land foraging and the pricier gear many campers pay hundredsif not thousandsof dollars for, all in the pursuit of bushwhacking primitivism. He is, he tells me over the phone from Edmonton, taking back camping for the people.

Beyond the appeal of his contentwhich combines man-vs.-nature survivalism, ASMR, and the camping scene from FubarCamping Steves approach is refreshing in part because camping itself can sometimes seem so rarefied. In a damning indictment, he compares contemporary camping to golf. It's turning into a pastime for the affluent, he says. The most wholesome form of camping is going out with a bedroll, and a fire, and a can of beans and sleeping out under the stars. We're paying to reserve campsites, which are just parking lots. We're buying RVs and campers that are just ridiculous. I saw a $300 backpacking tarp the other day at the camping store!

Like so many activities, campingroughly defined as lodging temporarily somewhere in the out-of-doorshas become a lifestyle. And a pricey one. A 2019 Global News story pegged the price of a basic outlay of geartents, sleeping bags, heavy-duty backpacks, bug balms, etc.and reservations between $978 and $1,333 CDN. The rise in popularity of more luxurious outdoor accommodations (the nauseatingly termed glamping) and expanded access to cell and wi-fi networks, have removed perceived barriers to entry, leading to would-be-campers across Canada (and North America) reporting an uptick in interest. Outdoorsy brands like Patagonia, North Face, and Arcteryx have grown from purveyors heavy-duty performance equipment to coveted dadcore lifestyle brandsallowing you to dress like a serious rock climber, even if you dont know a bowline from a belay. Where camping and camping-adjacent outdoor activities (canoeing, climbing, angling, etc) were previously conceived as a way to rekindle a relationship with nature, that relationship has become just another luxe commodity. (See also: the related phenomenon of #VanLife, which I have a hard time reading about without getting so annoyed that my heart goes arrhythmic.)

Theres this mentality where you think you have to spend so much money, says Wallis, its not camping unless you've paid to reserve your campsite, and you use your week of vacation a year, and get top-of-the-line stuff you only use once and then it'll sit in your garage forever. I'm probably part of the 1/1000th of a percent that has gotten their money's worth from sleeping in a tent. For Steve, its not about gear, or pricey base-layers (what the Patagonia merchants call T-shirts, as if drinking 35 Mooseheads in autumn at a friends parents cottage requires attire befitting the K2 Base Camp), or $300 technical tarps. Its foremost about getting out there, and just being nature, with whatever gear works. Its also, in many cases, about the thrill of doing so stealthily.

When hes not camping out on Crown Land or testing the resilience of all-weather tents in his yard, Wallis is sneaking around closed campsites, residential areas, and parking lots. He spent years in Victoria, BC living out of an RV which was both his vehicle and primary residence, camping in parking lots and off logging roads. He calls that phase, boondocking. It was a lifestyle not by choice, but necessity. He now lives in Edmonton with his wife (who never appears on-camera in his YouTube videos and is referred to, almost exclusively, as Beautiful Wife) and runs his own heating and gas company, Hunker Down Heating. But Wallis, as he explains, got the bug during his boondocking years. There's a risk to getting caught, he says. There's a game of cat-and-mouse, hide-and-seek. And you're getting a good deal, because you're parking for free for the night!

Wallis found YouTube about three years back. He was always aware of it, but figured it was a platform for goofy, viral videos, and not for personalities. When he camped out in -32C and posted the video, he saw an immediate response. I was getting a lot of comments from people who were interested in this kind of thing, and following along, he says. I ran with it from there.

Some people have accused him of basically cosplaying as homeless; of making a game out of being sleeping rough. He doesnt think so. In fact, he thinks his adventures give him a certain perspective. "Every morning where you dread going out to scrape off your windshield, someone has been sleeping out in that all night, he says. It's a big eye-opener. Unfortunately, in this weather, people do die every year, sleeping out there. It's a tough world out there for people who don't have a home.

Beyond the stunt-factor of camping in well-below freezing temps or hunkering down in a rented U-Haul in an airport parking lot, the primary appeal of the channel is Camping Steve himself. His videos are rack up plenty of comments from viewers saying stuff like, I dont know why but I cant stop watching. There is, undoubtedly, just something about him: an even tone to his voice, an ability to remain utterly unflappable even when security guards are hammering on his van doors, black bears are looming on the perimeter of his campground, or his jerry-rigged camping raft gets beached on the banks of an Alberta river. Theres even a pleasing rhythm to his videos, in watching him find a spot, set-up camp and crank a celebratory beverage (he calls this ritual Step Two, to the point that in recent videos he just straight-up refers to beers as Step Twos).

Some viewers have dubbed Wallis The Bob Ross of Camping. And it fits. Hes calming, reassuring, and just irrepressibly kind-seeming (He leans into this a bit, compiling a YouTube video playlist called Sleepytime Camping Mix.) Wallis isnt much interested in parsing the appeal that has led him to 165K subscribers and sees his videos regularly draw in 1,000,000-plus views. Whatever it is, I don't want to get the yips and over-analyze this. I just want to keep doing what I'm doing.

Its that candourthat utter lack of guilethat drew me into Wallis own cozy YouTube warren. (Also: he has a nice a nice smile and he reminds me of my friend Mike.) Shows like Alone and Naked and Afraid are by-and-large fun watches because you get to see people who style themselves as hardened lone survivors absolutely suck at all the feats of high-level outdoorsmanship they insist theyre amazing at. Steve Wallis camps under Walmart tarps and cooks corn niblets in a can and slugs back belts of Wisers not to prove something to himself or his presumed viewership, but because he genuinely loves it. He likes the breeze and bedrolls, the nip of the autumn air, and the canopy of stars. He also really likes upping the ante. He tells me hes noodling with the idea of building a large treehouse thing in a future video. These are things I wanted to do as a child, but never could, he says. I'm thinking maybe a hovercraft of some kind. A camping hovercraft.

A camping hovercraft. Now thats some serious marrow-sucking. Thats camping with Steve.

Follow John Semley on Twitter.

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This Albertan YouTuber Is the Bob Ross of Stealth Camping - VICE

Rick & Morty: How It Evolved From A Back To The Future Riff (& Where It Might Go) – Screen Rant

On its surface, Rick and Morty is a gonzo animated comedy about a mad scientist and the slightly dopey grandson he drags along on his adventures through multiple dimensions. In regard to the shows earliest episodes, thats not an unfair description. Much of season 1 is filled with pop culture hot takes, toilet humor, and even a few uncomfortable gags about sex abuse. Right from the off, though, there was something genius bubbling under the surface. Early-adopting fans could see it, and some of the first episodes for example, Lawnmower Dog (season 1, episode 2) and Meeseeks and Destroy (season 1, episode 5) managed to showcase some of the themes and ideas that would make Rick and Morty great.

Related: Is Rick & Morty On Netflix, Hulu Or Prime? Where To Watch Online

It was in season 1, episode 6, Rick Potion #9, that the show found itself. There is a caveat here: the shows seasons arent written in airing order, so there is room for debate on the quality of what comes before and after this episode. What makes it seminal, though, is that its the first episode of the show to bring all of Rick and Mortys signature elements together. It gave the show a core philosophy while developing its characters in unexpected ways. Theres also an astoundingly original twist and plenty of sex jokes.

Rick Sanchez, the scientist of the shows title, embodies nihilism. He believes that because the cosmos is endless, filled with infinite realities and inconceivable time, the life of an individual is essentially meaningless. There is no true right or wrong, and self-preservation is the only law. When the show begins, he is the only character in the shows core family his grandson, Morty; granddaughter, Summer; daughter, Beth; and son-in-law, Jerry who subscribes to this reality; as a result, Rick does some terrible things to his family,whilethey, in turn, are all thoroughly caught-up in their mundane suburban lives.

The nihilism in Rick and Morty is key to the show for two reasons. First, its contagious. At the end of Rick Potion #9, Rick and Mortys version of Earth is overtaken by mutant beings accidentally created by Rick. Rather than fix the world, they are forced to find an alternate reality where they have both died, so that the original Rick and Morty can seamlessly continue their lives with another version of their family. Not only is that a great twist, it serves as Mortys first taste of philosophical horror. His life hasnt been changed in any way, but technically, all the people around him are strangers.

Two episodes later (season 1, episode 8, Rixty Minutes"), Morty seems to have embraced nihilism. When confessing his true identity to the alternate Summer, he tells her, Nobody exists on purpose, nobody belongs anywhere, everybodys going to die. Come watch TV. Morty has accepted meaninglessness, although his words say more than that.

Related:Rick And Morty: Annie's Bleak Fate Explained

Nihilism does spread to other members of the Smith family throughout the show, but theres a wrinkle. Rick and Morty isnt simply touting nihilism as the one true philosophy. Its more interested in commenting on how that belief system affects individuals. In the first scene of the shows pilot, Rick is belligerently drunk and he stays that way through most of the episodes. Hes a suicidal addict who abandoned his daughter for most of her life, and hes immune to self-reflection because of his nihilism. When Morty says, Come watch TV, hes taking a totally different approach. He places value on everyday activities and his relationships precisely because they are the only things that hold meaning.

As the show progresses through its second and third seasons, some contradictions to these ideas do come up. The attention to character development, though, makes Rick and Morty even more interesting.

It would be fair to criticize Rick and Mortys earliest episodes as having one-dimensional characters. Rick is a tortured genius, Morty is a dim-witted teen, and Jerry is an aloof fool. The female characters arent much deeper: Beth wants nothing more than her fathers approval, and Summer is a sarcastic drama queen. Its to the writers credit that these shallow traits become much denser as the show goes on, as Beth and Summer become Rick and Morty'sunsung heroes.

Theres some debate as to whether Rick learns or grows as a character. While his philosophy and abrasive exterior dont change much, spending time with Morty has an effect. In the climax of the season 2 premiere (A Rickle in Time), Rick nearly sacrifices his life to save Mortys. This sets him apart from any alternate Ricks, who treat their own versions of Morty as expendable.

Related:Rick & Morty: The Best Smith Family Team-Ups

As Rick grows softer, Morty gets tougher. In season 3, episode 6 (Rest and Ricklaxation), Mortys worst characteristics are stripped away and become a sentient, toxic version of himself. Toxic Morty is almost identical to the one in the shows early episodes. He is jumpy, neurotic, and subservient. This is vastly different than the Morty who casually and skillfully defuses a neutrino bomb in the same season (season 3, episode 4, Vindicators 3: The Return of Worldender).

The other family members change, too. Each finds independence in his or her own way whether through divorce, resiliency, or pure physical strength. The dynamics of the Smith family are constantly changing in incremental ways, such as Jerry's redemption arc in season 3.

Rick and Morty doesnt take itself too seriously. For all its commentary on philosophy, society, and family, it is a genuinely funny show. There is plenty of frat boy, stoner comedy though it has wisely steered away from the misogynistic, anti-PC jokes of its early days but that isnt the only audience that can appreciate it. Ricks blunt one-liners border on shock comedy, Mortys ill-timed stutters feel improvisational, and Jerrys incompetence is almost slapstick.

Rick and Morty doesnt have a signature style because it doesnt want to. Repeating jokes about nazis in Rick and Mortyare undercut with belches and nudity. Sometimes Rick talks directly to the audience in a way that acknowledges hes nothing more than a cartoon character. The show is fun to watch because its constantly changing its approach to comedy. While its fans obsess over the nuance and depth, the show itself is refreshingly uninterested in all that.

With the recent announcement that Rick and Morty has been renewed through Season 10 (Season 4 is currently on a midseason break), there has been a lot of speculation about what will come next. That is a huge order of episodes, and for a show so irreverent, there is no telling. If the first episodes of Season 4 are any indication, Rick and Morty is ready to embrace its silly side. Its writers feel like theyre teasing the fans who dig into the weeds of its philosophy. This trend of bonkers but still clever episodes could continue, though it surely cant sustain six-and-a-half more seasons.

Justin Roiland and co-creator Dan Harmon are smart storytellers, and they know that shows have to change and evolve. Even if theyve grown bored with all the philosophizing around Rick and Morty, its characters will undoubtedly continue their satisfying arcs. As Rick reluctantly allows himself to get more attached to his relationships, Morty will continue to come of age as a smarter, more capable teenager. The context of that growth could go anywhere; the possibilities of the Rick and Morty universe are inherently limitless. If Roiland and Harmon can keep its characters on the rails, it will remain one of the most exciting shows on television.

More:Every Movie Reference In Rick & Morty's Episode Titles

Why The Dark Knight Recast Katie Holmes As Rachel Dawes

Graduate of New York University's Dramatic Writing program. Self-published novelist and podcaster. Passionate about all film, from the Halloween series (even the bad ones) to French New Wave. Enthusiastic about travel, hiking, and creating music playlists that are way too long.

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Rick & Morty: How It Evolved From A Back To The Future Riff (& Where It Might Go) - Screen Rant

Urban Dictionary: Nihilism

Contrary to popular definitions, Nihilism is not synonymous with cynicism or despair. Instead, Nihilism is a worldview in which one believes only in what one's observations and experiences seem to prove true, and that which can be otherwise proven true. That said, Nihilism varies according to the nature of the individual nihilist, but there are a few key ideas which are kept by nearly all of them: 1. The beginning of the universe was, within certain parameters, a basically random event, and the same holds for all events occuring since. It follows, then, that final purpose in things is false. Life, then, is an end-in-itself.2. There exists no absolute truth regarding the value of any deed over another, such as right vs. wrong. Value systems, ethical codes, etc. are thus of no use to the Nihilist, except if they serve his best interests, increase their quality of life, or if they simply fall in line with what behavior would come naturally.3. From the above it follows that responsibility, obligation, and the like are also falsehoods. Nihilists are thus inclined to ignore or sneer at societal norms and conditioned mentalities.4. The first priority of every nihilist is his own well-being, satisfaction, and survival, and every action is ultimately done in the name of these things. However, he does not consciously pursue these ends; instead, he acts upon what feels natural and makes sense to him, and these naturally result. However, the above assumes that the Nihilist is in unity with himself, and possesses an undamaged psyche. In reality, some people are self-destructive by nature, and, if they took up a Nihilistic worldview, would seem to have a death-wish as the motive behind their actions. Since self-destructive individuals are common in modern society, this is probably how Nihilism has come to be seen as another word for despair.

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Urban Dictionary: Nihilism

What’s the Point of Being a Good Humanist in a Mucked-Up World? – Patheos

Lets begin with a story. Its the one where everythings going wrong: zoonosis has triggered a massive viral outbreak, and associated spike in racism; Brexit has gone into effect, emboldening further racists; democratic checks-and-balances are being denied in plain view in the U.S. political system; citizen monitoring and related forms of state control are on the rise in genocide-perpetuating China; the consequences of climate change are being felt the world over in extreme natural events and increasing refugeeism; populist presidents are still thriving through the implementation of brutal initiatives in a wide range of countries; indigenous activists are still dying in their on-the-ground attempts to help protect natural resources; our food systems are making us unhealthier; and if obesity or opioids dont kill us our loneliness epidemic will.

When I talk about humanismthat is, when I talk about the need for spiritual and secular people to put aside cosmological differences to focus on a shared interest in empowering human beings with empathy and scienceIm making a pretty darned big leap in presuming that we have any agency at all.

Because some days itreally doesnt seem like we do, does it?

We cut out single-use plastics and then read articles telling us that our substitutes might be making matters worse.

We strive to eat less-than-perfect-looking groceries, then discover that such trend cycles can create different forms of waste, when the food industry already has perfectly good uses for ugly vegetables.

We recycle our hearts out, then find out things about the recycling industry that make us doubt if its good enough.

We reduce ourmeat intake, but thentake a vacation worse than many peoples entire annual CO2 footprint, meat and all.

And are enough of us going to stop reproducing? Ever?

Is it reasonable, is it desirable, is it attainable, to be a good humanist in such a staunchly mucked-up world?

To be clear, after all: Therearealternatives to humanism, both spiritual secular. If there werent, to say one is humanist would be meaningless, on par with saying I am human. (Which Im certain most of you are.)

Theres existential nihilism, of course: the idea that life has no intrinsic meaning and nothing we create has meaning, either. For a spiritual nihilist, this looks like if you are not in favour with our god, your life is meaningless and probably forfeit. For an existential nihilist, this looks like theres no intrinsic meaning in the cosmos and no point to contriving a sense of meaning during my time alive. Do as you will until you die.

Then theres worldly detachment, Buddhist-flavoured or otherwise, in which one seeks a state of reduced investment in what the world is and isnt, aspiring simply tobe without striving for any greater insight or value in the time youve spent alive.

And then theres my personal favourite, absurdism. As in Albert Camuss famous summation of the precept, il faut imaginer sisyphe heureux (One must imagine Sisyphus happy), absurdism tells us to live with full awareness that life is meaningless without either killing ourselves or falling in with dishonest philosophy, instead being content with the struggle to hold the line between these options. Why? Because there and there alone, in ourchoice to be content with an existence we do not control, do we have any agency in the cosmos at all.

So, sure, we could be absurdists instead. (I often long to be one after certain news cycles.)

But thereis a critical difference between absurdism and humanism, and it lies in that notion of aspiring to contentment.

For an absurdist, thats it. Lifes a grand cosmic joke: revel in it!

For a humanist, though, we might ask for a broader range of responses.

We might say, that is, il faut imaginer sisyphe insatiable: insatiable for new knowledge, insatiable for new experience, insatiable for self-improvement.

Because who says he cant learn to sing while pushing that rock? Who says he cant holler at other denizens of Hades and ask them how the underworlds treating them? Who says he cant offer counsel to them in their own suffering, and share his own low spells to receive counsel and support from them in turn?

(And then, in more tragic cases, weve sometimes achieved that control by hastening that end along through war or other forms of mass suicide.)

Harder, far harder, is to invest in the struggle itself. This isnt the same as being hopeful (though youre welcome to feel hopeful, too, if youd like). This isnt about finding new and better platitudes to replace the old, and clinging to any sense that if we just try a little bit harder, a little bit longer, everything will work out in the end.

Secular humanists: Weknowit wont. The end is oblivion. Thats as much as anything ever works out in the cosmos.

So, no, this isnt about optimism, per se. This is more about recognizing when you are tired, and fed up, and cynical, and hurt, and wounded, and angryall conditions in which it seems reasonable just to stop botheringand then to make a concerted effort to keep bothering anyway.

Not because of some promised better world, spiritual or secular.

And not because we truly believe that racism, sexism, xenophobia, and other forms of social persecution will be solved in our lifetimes.

But because there is a gravity to saying, Everythings mucked-up and Im sticking with it anyway. Say it to yourself. Try it (or more forceful variants) out. Theres a conviction to it, isnt there? A grounding assertion of inner will.

Its our ultimate test, my friends. Can we get over ourselves, get over our egos, get over our hurting pasts and our grinding presents, get over all the people who ever wronged us and all the things we can never fix, long enough to be able to say, Existence does not have to be happy or hopeful to be a space where meaning is made?

After all, if there were a great big red button we could press to terminate everything simultaneously, okay, finethen wed be in a situation where it would be worth haggling over how much suffering is too much.

But we dont, so we arent. Instead we have a world where were all linked to one another, and our collective suffering rises and falls with one another. Much as we might want to live with perfect detachment, then, and much as we might want to affect absurdist humour through it all, the day-to-day reality of our interactions with other people and their struggles is always going to pull us back in to the agony of meaning-creation in human society. (I mean, unless youre already living in a remote cabin somewhere, disconnected from the entire ecosystem of human struggle? In which case, why the heck are you reading this?)

And it isagony, because meaning-creation is not easy, and its not always successful. We despair, we lose, we fail. Routinely. Daily, even. But even if were not high enough in the chain to make sweeping and sustainable changes to how much other people suffer, we can strive to alleviate proximate complaints: sometimes through aid; sometimes through presence; but most of all, by learning to look at our circumstances and remind ourselves that there is as much value in this form of existence as there is in any other, rich or poor, sea-slug and moutain-lion alike:

Namely, as much as we choose to invest in its growth.

So invest well, fellow humanistsin new knowledge, new experiences, new approaches to self- and communal improvement based on the best new intelall while expecting no greater reward than this:

That you invested in your existence for as long, and as meaningfully, as you could.

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What's the Point of Being a Good Humanist in a Mucked-Up World? - Patheos

Boston Is Losing Its Children Whats At Stake, And What Has Already Been Lost – WBUR

Ive been thinking a lot lately about Alfonso Cuarns Children of Men. Set in a dystopian future where infertility has given the human race a literal expiration date, the movie is about what happens to a society when the future fades out of sight and people embrace nihilism.

Children, the movie seems to suggest, are theraison detreof our constant struggle to make life better. Even if you dont have kids of your own as many of my generational peers do not and will not its possible to be humbled by having kids around. Knowing that there are always more little humans on the way can inspire people to think beyond themselves and do things that benefit humanity. It can be a tonic for loneliness, depression and other afflictions that ail us.

Try to imagine a world without laughter echoing from playgrounds. I cant think of a time in my life where this soundtrack wasnt playing somewhere I cant imagine life without it.

Except now, as a resident of Boston, I have to.

A newstudyby The Boston Foundation offers a chilling vision of Bostons future, so far as kids and families are concerned. As the Boston Globe housing reporter Tim Loganwrote, Boston has grown by about 100,000 people [in the last two decades]. But it has about 10,000 fewer school age kids. Where have all the children gone? The answer, according to the study, is that families with kids are being pushed out of Boston, into less expensive (but still gentrifying)suburbs, at such a seismic rate that in the near future, children might not have a place here.

This might sound hyperbolic, but its the simple conclusion of The Boston Foundations report, and its substantiated by data.

School enrollment rates in Boston have plummeted since the mid-20th century. Roughly 20,000 students mostly from low-income households have been lost. But the total number of children living in Boston, irrespective of their school enrollment, is far more disturbing. Of the 700,000 or so people living in thecity, only 75,000 of them are between 5 and 17 years old. Thats barely 10%, and its a staggering 43% drop from the number of children living in Boston50 years ago,in 1970.

Lately, theres been so much reporting about theforcesdrivingthousands of Boston residents into the suburbs. But lets remind ourselves of the obvious: the housing werebuildingis mostly designed for well-off young professionals,publichousing and even housingvouchershave been painfully under-used in recent decades, and the decision-making class of Boston cant seem to reconcile its fealty to the markets, with the idea that cities should be more than giant business districts.

Consider the Seaport, a new neighborhood with plenty of mixology bars and co-working spaces, but not many of the things that families with children need, such as public parks, libraries or even a grocery store. As the Globes Spotlight teamremindedus in 2017, the Seaport project was a chance to create a neighborhood for all Bostonians, including children. Some $18 billion in taxpayer money was funneled into the Seaport, on the premise that the new neighborhood would be a hub for innovation and an economic development boon for all of Boston. Instead, its become home to luxury apartments and one of the wealthiest and whitest neighborhoods in the city.

There are severallocalexamples of the development and gentrification thats turning Boston into a childless city,while also hurting surrounding communities too. There's thefactthat many of the new apartments and condos built recently are either sitting empty, or being used as havens for investors.

The Boston Foundations report is not a warning. Itsreminderthat the epidemic displacement of families ...has been happening here in Boston for decades.

Lesser known are the ripple effects that the Boston housing affordability crisis is having for "gateway cities." Just one example of many: in Lynn a youth education center is being replaced by a luxury apartment complex that will boast, among other amenities, an indoor golf simulator.

Its Bostons turn to decide what we do with this report, and how we reckon with the future that it predicts.

As a millennial without any kids, paying below-average rent but living in a place that would not be remotely suitable for a family, Ive tried to offset my own impact on the housing situation by learning (andwritingabout)local activist organizations such as City Life/Viva Urbana. That organization has been fighting to protect families from evictions and to advance more equitable housing policies, such as municipal rent control and expanded tenant protections.Groups like City Life/Viva Urbana are showing our leaders what needs to be done to protect and sustain families who, against the odds, are still living here. Their voices and ideas should be amplified.The Boston Foundations report is not a warning. Itsreminderthat the epidemic displacement of families that we've seen in cities likeSan FranciscoandSeattlehas been quietly happening right here in Boston for decades.

It doesn't have to be this way. Boston is a city where structural forces like capitalism and racism have sometimes been tempered by people with a genuine commitment toward social responsibility. This is an age-old battle that's been fought in places such as the Dorchester Youth Collaborative, which camecloseto shuttering in 2017 due to funding shortfalls, or the state's Department of Conservation and Recreation, which is currently in questionby Governor Baker's latest budget.The green spaces in Boston where the public can still roam are a reminder of what's at stake.

Unless Boston aggressively confronts our housing and transportation crises head on, the playgrounds that have not yet been replaced by craft beer gardens and bocce ball courts could one day become empty and silent.

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1917 Is a Best Picture Frontrunner and That’s Frustrating | CBR – CBR – Comic Book Resources

After a long and dramatic Oscar race, it looks as if Sam Mendes' World War I adventurestory1917is poised to take home the coveted golden statue as Best Picture. The film started its awards streak with a win for Best Picture - Drama at the Golden Globes, which, at the time, was considered a surprise. As awards season progressed, however,1917 no longer seemed likean upset of a choice for Academy voters, as the acclaimed feature took home the top Producers' Guild Award as well as the Directors' Guild of America Award.

However, theAcademy Awards have a longstanding reputation for recognizing war filmsin lieu of other genres of filmmaking.1917, despite its impressive technical achievements, adds little to the pantheon of Oscar-winning cinema about the horrors of war.It's an unfortunate turn of events for the other nominees, which arguably broke bolder narrative ground than a gorgeously shot but simply told World War I story. Furthermore,1917 doesn't challenge the viewers' ideas on war in the same way other films released this past year attempted to broaden audiences' horizons on morality, violence, and socio-political issues.

RELATED: Are Superheroes Too Bland to Ever Receive Oscars Love?

TakeJoker, for instance, which completely reinterpreted the limits of a comic book movie. While the narrative of1917follows familiar war movie beats,Joker seeks tomake the audience feel uncomfortable aboutpreconceived notions of the title character. While it may follow a previously established plot formula taken fromTaxi DriverandThe King of Comedy, the film argues that comic book stories don't have to be limited to blockbuster action sequences and colorful, heroic adventures. Critics of Jokerargue that the movie is too dark and cynical, completely failing to address its message in favor of reveling in nihilism. But at least director and co-writer Todd Philips attempted to challenge viewers' ideas of morality andthe corroding effects ofcapitalism, however bleak and frustratingly pointless that depiction is.1917, on the other hand, merelytips its hat to the British troops for their sacrifices.

Another entry up for the big award is Jojo Rabbit, which also works as an apt comparison to the flatness of1917.Writer and director Taika Waititi made a bold choice in looking at World War II from the perspective of the antagonistic side of the war, and received a fair share of controversy forusing the Holocaust as a backdrop for a comedy. However, his intention was to use satire to depict the sheer absurdity of hate. The fact that the film takes place on the German home front means that it can also show the multitude of German citizens living under the Nazi regime, whether they were reluctantly participating in the war or actively resisting it. Compare this to the German soldiers in 1917, who arenameless murderers with no sign of remorse or moral compass. There's a debate to be had about how responsible theexecution ofJojo Rabbit is, but it should be commended for addressing complex wartime issues.

RELATED: What Joker & Parasite's Screen Actors Guild Wins Mean For the Oscars

In fact, the influence of social pressures on the perception of morality seems to be a common trend among most of the Oscar nominees.Parasite is a fresh take on class issues thatsuggests the tensions between the haves and the have-nots are not so clearly defined. The Irishmanpresents a protagonistat a moral crossroads as he tries to find personal and financial success within his dark, criminal business. Marriage Storytakes a closer look at another type of war altogether, allowing the audience to sympathize with both sides of a gendered, interpersonal conflict in the form of divorce proceedings. All of these films push the boundaries of cinematic storytelling through their complicated characterizations and topical themes.

Sam Mendes' epic is a Hollywood blockbuster taken to stressful and bombastic heights, but it's still a Hollywood blockbuster that conforms to a specific formula. In fact, the plot almost resembles a superhero movie or Call of Duty video game in its straightforward telling of a quest to reach a specific location to deliver a message. The one-take technique allows the members of the audience to feel as if they are experiencing the horrors of war on a personal level. However, 1917 doesn't make a compelling statement other than how hellish war is, a message that countless war films in the past have meticulously covered.

To be fair,1917is a highly ambitious technical masterpiece. For all the war movies that have passed through the annals of the Academy, none has ever felt quite as immersive as this one has. Cinema is ultimately subjective, so one idea of "revolutionary filmmaking" may look different than another. Still, the ideas of1917feel oddly conventional compared to the more contemporary messages of the other Best Picture nominees. Even Once Upon a Time in Hollywood, a film that celebrates a bygone era, follows an unconventional narrative structure. In a field full of stories that passionately address current issues,1917stands out as an ode to past traditions.

NEXT: Why the Oscars Snub Disney's Blockbusters -- Repeatedly

Thor, Herald of Thunder, Proves Just How Strong He Really Is

Tags:feature,Joker (film),1917

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1917 Is a Best Picture Frontrunner and That's Frustrating | CBR - CBR - Comic Book Resources

Rachel Maddow on her critics: Your hatred makes me stronger. Come on! Give me more! – The Guardian

Rachel Maddow, the US TV host, has a message for her critics: Bring it. Your hatred makes me stronger. Come on. Give me more. Give me more. I love it!

That is just as well, given the way she has polarised viewers in the past few years. Maddow, 46, has presented her primetime show on MSNBC, a 24-hour cable news channel, on weeknights since 2008. If most cable news and social media is fast food, she tries to rustle up a three-course meal: sensible, sober, sometimes painfully detailed and replete with oblique references to half-forgotten politicians of yesteryear. Since Donald Trumps rise in 2016, however, The Rachel Maddow Show has become a nightly safety blanket for many progressives who identify with the resistance.

Her figure has ascended, in the liberal imagination, from beloved cable news host to a kind of oracle for the age of Trump, the New York Times Magazine noted recently, describing her show as the gathering place for a congregation of liberals hungering for an antidote to President Trumps nihilism and disregard for civic norms.

The view from the right is rather different. Maddow is demonised as an avatar of the liberal elite and Trump derangement syndrome. Even ideological fellow travellers worry that she is strident and shrill. She dedicates her new book, Blowout, which explores the pernicious influence of the global oil and gas industry, to the bots and trolls, all of you, with love.

There is, of course, much more to Maddow than her screen persona. A practising Catholic as well as the first openly gay host of a US primetime news programme, she has suffered cycles of depression all her life and enjoys fishing, shooting and reading Robert Harris historical yarns. She is also, it turns out, a lot of fun.

At a receptionists desk outside her understated office in New Yorks art deco Rockefeller Center sits a decaying lifesize waxwork of the former Republican president Dwight Eisenhower. Inside, a Russian doll bearing Vladimir Putins face rests on a bookcase. Crutches are propped up in a corner as a talisman she broke her ankle last year in a fishing accident.

With bookshelves overflowing the Mueller report, examining Russian interference in the 2016 election, lies on a crate below and ideas scribbled on whiteboards, it is the den of a promiscuous intellect. Maddow, dressed in a black jacket, turned-up jeans, camo trainers and her trademark black-rimmed specs, spots my voice recorder a vintage Tascam device that resembles a Taser and snaps a photo of it with her phone, lest it be useful to her team.

The media will be on trial in Novembers presidential election. Many observers fear a repeat of 2016, when Trumps vulgar antics gained endless air time, pundits were insufficiently critical and even serious newspapers appeared to treat baseless allegations against Hillary Clinton as if they were equivalent to Trumps financial and sexual transgressions.

The day after we speak, Maddow demonstrates another way of working and gains the biggest ratings win of her career when her interview with a key figure in Trumps Ukraine scandal pulls in 4.5 million viewers, beating all the competition. Her explosive conversation with Lev Parnas, an associate of Rudy Giuliani, Trumps lawyer, is a timely riposte to the claims that she is too partisan, polemical and fixated with international conspiracy theories. It makes the political weather for several days.

I think part of the reason Trump got so much earned media during the 2016 campaign is that he was so transgressive, Maddow says. It was a relatively rational newsworthiness assessment to put a camera on him at all times, because there was a reasonably good chance he was going to do something shocking, wrong, transgressive or unbelievably disruptive.

Getting a show on the air is like merging on to the freeway at 80 miles an hour at night in a storm with no headlights

But that also dovetails with the question of whether or not his falsehoods and reversals and self-contradictions and professed ignorance got the critical coverage that they deserved. When nobodys expecting you to tell the truth, you telling a lie isnt news. If Barack Obama got up and said something that was proveably false or maybe not the whole truth, youd have the whole media, not just Fox News, going crazy for a month at a time. Donald Trumps mendacity has never been notable. Its just been baked in.

Trump made more than 16,000 false or misleading claims in his first three years as president, according to the Washington Post and that number is likely to accelerate as the election hots up. But he is undeniably good for ratings and clicks. Media outlets will have to choose whether or not to broadcast his campaign rallies and interview his surrogates unfettered, as well as how far to go in editing, fact-checking or simply no-platforming.

We went through our own transformation fairly early on when I realised that I couldnt put Trump administration officials on the air as guests because they did not tell the truth, says Maddow. Having to clean up after your guest is a sort of fool me once thing, like: When I told my audience that you were somebody worth listening to because you had something to contribute to our understanding of the world, I was wrong. Ive goodnighted you. I wont invite you back.

She adds: I can play the tape of the president saying something thats not true and then clean up after him but then Ive also just played the tape of him saying something not true, and maybe thats more compelling to you and more of what you remember than the part where I corrected it.

I feel like theres a responsibility level there. So were not going to give people a platform who are lying to the public. But Im assuming everybody else in the media is going through that same thing.

Observers of all political stripes have taken Maddow to task for her coverage of Trumps alleged collusion with Russia. Presidential cheerleader Sean Hannity, whose Fox News show airs in direct opposition, has called her the chief conspiracy theorist. In a critique of her handling of a 35-page dossier on the subject compiled by the former British intelligence officer Christopher Steele, Erik Wemple of the Washington Post wrote: She was there for the bunkings, absent for the debunkings a pattern of misleading and dishonest asymmetry.

But Maddow, whose new book was inspired by Putins motives for attacking US democracy, describes some of the criticism as dishonest and makes no apologies for being obsessive, especially since there is every prospect of Putin trying again this year. Russia interfered in the 2016 election to try to elect Donald Trump, and Donald Trump got elected and he is weirdly and irreversibly supplicant toward Russia and Putin. Like, OK, Im going to cover that, she says. I dont care what anybody says about me. I dont play requests and I dont worry about the criticism. If we get something wrong, Ill correct it, but, in the absence of that, the criticism for focusing on real news stories that bother people thats what I get paid to do.

You have to grow a thick skin, she says. Getting a show on the air, even all these years in, is like merging on to the freeway at 80 miles an hour at night in a rainstorm with no headlights and no windshield wipers and a bunch of semi-trucks coming up right behind me. Somebody telling me that they dont like the colour of my car? I am not focusing on you! Ive got a job to do here.

That freeway sounds terrifying and yet also an adrenaline rush. The Trump presidency is a liberals worst nightmare and a dream for many journalists. Jim Acosta, the chief White House correspondent at Maddows rival network CNN, has said: Im having the time of my life right now. This is the biggest story of my life. Im like a kid in a candy store. So, despite the daily horrors, is Maddow secretly enjoying this?

Civil rights arent a given. Theyre asserted and protected or assailed

I dont know if enjoy is the right word, but I do have the best job in the world, she says. I talk to a lot of people who feel strongly about whats going on in the world, whether theyre in public affairs or theyre just concerned citizens who pay attention to whats going on. People say: Dont you find it incredibly demoralising? How do you think about all this stuff all day? I feel the opposite. If youre at all engaged with whats going on in the country and in the world, you have to be concerned. But my job, what they actually pay me to do, is to read the news all day long and do original reporting and try to figure out what is and isnt reliable information. I feel like Im doing mental health work all day long.

Maddow, who grew up in a small town in California, has previously worked as a labourer, a messenger, a waitress and a bucket washer at a coffee-bean factory. I am not a planner. This definitely wasnt like the thing that I set my mind toward and then I put together all the pieces to get there. I had much more of a drunken stumble through my career than you might expect, but now that I have landed here, I value it.

She came out at Stanford University by posting an open letter inside every bathroom stall in her dormitory announcing that she was a lesbian, an incident that was reported in the student newspaper. Classy, right? I was 17. Why did we do anything? I dont know. I havent thought about it in a long time. I think that I was very full of myself and thought that this was a very important piece of news that everybody needed to have. And I also liked needling people and confronting them in ways that I thought would be both funny and self-aggrandising. I dont look back on that with particular pride.

Her conservative Catholic parents were stunned. I didnt handle it well. I wasnt very respectful and didnt have much emotional peripheral vision in terms of the way that my actions would affect others, which I think was a product both of my ego and self-regard and where I was in my own development at that time. They had a really hard time at first and we had a few rocky years.

But now my parents could not be more supportive. Im Catholic and I come from a Catholic family and the churchs teachings on this have been a factor. But, ultimately, where weve ended up is at the best possible place and I am super-blessed to have the support of my whole extended family.

Maddow has been with her partner, Susan Mikula, a photographer, for two decades. Counterintuitively for a liberal icon, their first date involving throwing tomahawks and firing guns at a Ladies Day on the Range sponsored by the National Rifle Association, of which Mikulas sister is a lifetime member.

Maddow, who does not own a gun, says cheerfully: Going to gun ranges is fun. Have you ever done it? You should go. You go and you pick which gun you want, you pay to use the time and they show you how to use it and you learn about the gun and then shoot a target. And then you leave the gun there and you go home.

Shooting sports is, I think, an interesting way to demystify guns, particularly if youre engaged in reporting on or commenting on the politics and culture around guns stuff. I dont have any problem with that at all. Its super-fun. Susan, it turns out, is a really good shot, which was a shock.

It says something about the times that Maddows ease with guns is more shocking to some than her sexuality. The CNN anchors Don Lemon and Anderson Cooper both came out almost a decade ago. Same-sex marriage was legalised in 2015. Now Pete Buttigieg, the former mayor of South Bend, Indiana, is bidding to become the first openly gay US president. Nothing, however, should be taken for granted. Under cover of myriad distractions, the Trump administration is attacking gay and transgender rights.

Maddow reflects: Civil rights and social justice issues often advance in what I describe as a sawtooth pattern of progress, where you take two steps forward, one step back. Its work that doesnt do itself. It has to be done by human beings who are working in a concerted way, in a way that is nimble and that appreciates the potential for backsliding and even backlash.

Thats the one piece of this that I feel like were learning more and more: that civil rights arent a given. Theyre asserted and protected or assailed.

Blowout by Rachel Maddow is published by Bodley Head. To order a copy for 17.60 (RRP 20), go to guardianbookshop.com or call 0330 333 6846. Free UK P&P over 15; online orders only. Phone orders minimum P&P of 1.99.

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Review: Be Here Now at Everyman Theatre. Seizures, sanity and the smell of spring – DC Theatre Scene

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What if happiness was not only a choice, but a side effect?

Thats the intriguing premise of Deborah Zoe Laufers play Be Here Now, which she also directs with depth and quirky humor at Everyman Theatre in a production that brightens the gloom and gray of midwinter with a joyful reminder to live in the moment even when the only positive thing you can say about your day is that you are still above ground.

Bari (Beth Hylton, equally commanding as a wretch and a radiant being) is a fortyish professor working at an Asian tchotchke distribution center in update New York. She is unable to complete her dissertation on nihilism that will allow her to keep teaching so she is gift-wrapping ersatz Buddhas and kvetching about her life.

And Bari is a world-class grump. Even at the age of five, Bari seemed to be in the clutches of existential angst. Her long-time friend Patty Cooper (a tough-as-nails and tender Katy Carkuff) recalls that she once asked the kindergartener Bari if she wanted to play, and the tot replied I dont play with cretins.

Cynicism and perpetual poop bobbing in the cereal bowlthats Bari. You may wonder why people like Patty, who terms her friends state of mind as smug gloom, and her ebullient niece Luanne (Shubhango Kuchibhotla, who is the living embodiment of a joyful exclamation mark), put up with her. On the other hand, even Oscar the Grouch has pals on Sesame Street.

Not only does Patty tolerate her friend, but shes fixing her up with a relative, Mike (Kyle Prue), a reluctant genius who makes astonishing structures out of abandoned items. Their first encounter is sweetly awkward, as two damaged people with limited social skills try to connect, but keep saying and doing the most comically wrong things.

Then theres Baris headaches that are worsening to the point where shes having seizures and hallucinating. But her seizures are transformationalin the grips of a hallucination the world vibrates with color and light and joy and every moment is a miracle.

What turns out to be a brain tumor turns Bari into her best, happiest self. Bari bathes in these strange, good feelings and never wants them to end. But she also grapples with who she truly issomeone who believes that life is meaningless like a mopey teenager or someone truly alive for the first time in her life?

Will this aura of awesomeness dissipate when the tumor is removed and Bari returns to her former role, a merchant of doom?

Be Here Now closes February 16, 2020. DCTS details and tickets

Luckily, Laufers play doesnt give us the answers we, or Bari, seek, but she teases us with possibilities as well as toying with the idea of a thin line between genius-creativity and madness. As Daniel Ettingers carousel-like set reveals the settings of Baris revelations, so does the play show us the value of mining the meaning out of every moment.

The play and the production is also thankfully free of touchy-feely, Oprah-esque maxims about finding your aura, self-care and the zen of happiness.

Be Here Now reminded me of Kay Redfield Jamisons books about moods, madness and creativity or of insider artists (like those at Baltimores American Visionary Art Museum) who created the most rapturous, ecstatic art as oddballs but when treated with psychotic drugs or brain zapped with electricity, their artistic voices were stilled.

Is it worth treating mental illness or a brain condition if it silences your genius?

The play makes you think about those touched with fire and how they handle it. Mike, played with beguiling ineptitude by Kyle Prue, retreats from society after a tragedy and devotes his life with monkish simplicity to making art out of what others throw away.

Yet Mike also quivers with the same sensitivity to what is all around him as Bari does when she has a seizure. There is this quietly revelatory scene late in the play, when Bari is slumped on a bench fretting that her golden, syrupy moments of pure happiness are transient. Mike is half-listening, his senses trembling as he sniffs the air.

Do you smell it? he asks her in wonder and at first all she detects is cat pee on an abandoned lampshade.

Spring, Mike states. And they sit quietly and sniff a new season.

Be Here Now .Written and directed by Deborah Zoe Laufer .Featuring Katy Carkuff, Beth Hylton, Shubhangi Kuchibhotla, Kyle Prue .Set Design: Daniel Ettinger. Lighting Design: Harold F. Burgeee II. Costume Design: David Burdick. Sound Design: Sarah OHalloran. Fights/Intimacy: Lewis Shaw. Stage Manager: Cat Wallis. Produced by Everyman Theatre . Reviewed by Jayne Blanchard.

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Review: Be Here Now at Everyman Theatre. Seizures, sanity and the smell of spring - DC Theatre Scene

Free-Speech Case Over Patron’s Arrest At The Kansas City Public Library Finally Comes To An End – KCUR

A lawsuit stemming from the highly publicized expulsion of a Kansas City library patron from a public event nearly four years ago has drawn to an end after the judge ruled in favor of the lone remaining defendant.

On Thursday, U.S. District Judge Beth Phillips found for an off-duty police detective who arrested Jeremy Rothe-Kushel, a documentary filmmaker from Lawrence who sued the detective and 13 other defendants over the incident, which drew national headlines.

Rothe-Kushel claimed his First and Fourth Amendment rights were violated after he was physically restrained on May 9, 2016, following a lecture at the librarys Plaza branch by American diplomat and former Middle East envoy Dennis Ross.

The lecture, about President Harry Truman's recognition of the state of Israel, was organized by the Jewish Community Foundation and the Truman Library Institute. Following the April 2014 shootings that left three people dead at the Jewish Community Center and Village Shalom in Overland Park, there was heightened security at the event.

During a planned question-and-answer session after the lecture, Rothe-Kushel stepped up to the microphone and asked Ross a long, rambling question alluding to what he said was a history of state-sponsored terrorism by Israel and the United States.

Ross responded and Rothe-Kushel began arguing with him. At that point, the man in charge of security for the event, Blair Hawkins, began to physically remove Rothe-Kushel from the microphone. Hawkins was director of security for the Jewish Federation of Greater Kansas City, which had hired him following the 2014 shootings.

Video of the incident shows Hawkins grabbing Rothe-Kushels arm, telling him Youre done and attempting to remove him from the mic. Rothe-Kushel is seen yelling even as a second person approaches the mic to ask a question.

Rothe-Kushel was later arrested in the lobby after an off-duty officer hired for the event asked for his identification and he refused to give it. The librarys director of programming and marketing, Steven Woolfolk, was also arrested after he sought to intervene and prevent Rothe-Kushels removal.

Woolfolk was later charged with obstruction, interfering with an arrest and assaulting a police officer. After a day-long trial in September 2017, a Kansas City Municipal Court judge acquitted him of all three charges.

The actions taken by the officers sparked outrage among civil libertarians and were condemned by the librarys then-executive director, R. Crosby Kemper III, who said the officers had overreacted.

Rothe-Kushels lawsuit named 14 defendants, including officials of the Jewish Community Foundation and the Truman Library Institute; the off-duty policemen involved in the incident; Kansas City Chief of Police Rick Smith; and members of the Kansas City Board of Police Commissioners, including then-Kansas City Mayor Sly James.

Rothe-Kushel later voluntarily dismissed his claims against the members of the police board and the off-duty officers, except for the detective who arrested him, Brent Parsons.

The claims against officials of the Jewish Community Foundation and Truman Library Institute had been dismissed earlier in the case although its not clear if they were dismissed because the organizations reached settlements with Rothe-Kushel or because of the merits of their legal defenses.

I can say that matters as to other defendants were concluded, said Arthur Benson, one of Rothe-Kushels attorneys. Thats all I can say.

Officials of the Jewish Community Foundation and Truman Library declined to comment or could not be reached for comment.

In her 12-page ruling Thursday in favor of Parsons, Judge Phillips found that Parsons had probable cause to arrest Rothe-Kushel for trespassing and for refusing to provide his identification.

She also found that while Rothe-Kushel had a First Amendment right to ask Ross questions, that right was not limitless: (H)e could not ask so many questions that other audience members were deprived of the opportunity, and he had no right to argue with Ambassador Ross (and no right to expect Ambassador Ross to engage in such an argument).

Finally, Phillips found against Rothe-Kushel on his claims of conspiracy to violate his civil rights, false arrest and conspiracy under state law.

Rothe-Kushel, reached by email, declined to say whether he had reached settlements with any of the defendants.

Fred Slough, another attorney representing Rothe-Kushel, said it was a serious wrong for Rothe-Kushel to have been removed and arrested. He said Rothe-Kushel would have complied with a request to leave the library.

Instead he was grabbed and manhandled in the middle of an exchange with the Ambassador that was not a disturbance, except in the sense that some in the audience audibly disagreed with its content, Slough said via email. The law does not allow such a heckler's veto of free speech.

Editor's note: This story was updated with Rothe-Kushel's comment.

Dan Margolies is a senior reporter and editor at KCUR. You can reach him on Twitter @DanMargolies.

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Free-Speech Case Over Patron's Arrest At The Kansas City Public Library Finally Comes To An End - KCUR

U.K. Police Will Soon be able to Search Through U.S. Data Without Asking a Judge – EFF

Law enforcement officials in the U.S. and U.K. have negotiated a deal that sells out the privacy rights of the public in both nations. For Americans, it will effectively abrogate Fourth Amendment protections, and subject their data to search and seizure by foreign police.

This is all going to start happening in a few monthsunless Congress does something to stop it now. Thats why were launching an action today, asking you to reach out to your members of Congress and tell them to introduce a joint resolution that could put a halt to the deal. If it isnt stopped, the worst parts of this deal will likely come standard on future agreements, and Americans will be subject to more and more searches by foreign police.

TAKE ACTION

Tell Congress to Stop the U.S.-U.K. Cloud Act Deal

The full text of the U.S.-U.K. Cloud Act Agreement was unveiled in November, and its just as bad as we thought it would be. We joined with 19 other privacy, civil liberties, and human rights organizations, and sent a letter to Congress going through the long list of problems with the first Cloud Act deal.

Some of the key problems with the U.S.-U.K. Agreement include:

In colonial times, the British military used general warrants to search through houses and seize property. This practice was part of what fueled the American Revolution, and formed the basis for the 4th Amendment to the U.S. Constitution.

Congress shouldnt let an executive agreement, negotiated behind closed doors, give away rights that have been enshrined in U.S. law for nearly 250 years.

TAKE ACTION

TELL CONGRESS TO STOP THE U.S.-U.K. CLOUD ACT DEAL

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U.K. Police Will Soon be able to Search Through U.S. Data Without Asking a Judge - EFF

FDA Continues Strong Support of Innovation in Development of Gene Therapy Products – FDA.gov

For Immediate Release: January 28, 2020

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patients cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.

In that spirit, today, the FDA is announcing the release of a number of important policies: six final guidances on gene therapy manufacturing and clinical development of products and a draft guidance, Interpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations.

The growth of innovative research and product development in the field of gene therapy is exciting to us as physicians, scientists and regulators, said FDA Commissioner Stephen M. Hahn, M.D. We understand and appreciate the tremendous impact that gene therapies can have on patients by potentially reversing the debilitating trajectory of diseases. These therapies, once only conceptual, are rapidly becoming a therapeutic reality for an increasing number of patients with a wide range of diseases, including rare genetic disorders and autoimmune diseases.

As the regulators of these novel therapies, we know that the framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field and further enable innovators to safely develop effective therapies for many diseases with unmet medical needs, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review; including how these products work, how to administer them safely, and whether they will continue to achieve a therapeutic effect in the body without causing adverse side effects over a long period of time.

One of the most important steps the FDA can take to support safe innovation in this field is to create policies that provide product developers with meaningful guidance to answer critical questions as they research and design their gene therapy products.

The six final guidances issued today provide the agencys recommendations for product developers on manufacturing issues and recommendations for those focusing on gene therapy products to address specific disease areas. The six guidance documents incorporate input from many stakeholders and take a significant step toward helping to shape the modern structure for the development and manufacture of gene therapies. The agency is issuing this suite of documents to help advance the field of gene therapy while providing recommendations to help ensure that these innovative products meet the FDAs standards for safety and effectiveness.

The scientific review of gene therapies includes the need to evaluate highly complex information on product manufacturing and quality. In addition, the clinical review of these products frequently poses more challenging questions to regulators than reviews of more conventional drugs, such as questions about the durability of response, and these questions often cant be fully answered in pre-market trials of reasonable size and duration. For some gene therapy products, therefore, although they have met the FDAs standards for approval, we may need to accept some level of uncertainty around questions of the duration of the response at the time of marketing authorization. Effective tools for reliable post-market follow up, such as post-market clinical trials, are going to be key to advancing this field and helping to ensure that our approach fosters safe and innovative treatments.

The draft guidance on interpreting sameness of gene therapy products under the orphan drug regulations provides the FDAs proposed current thinking on an interpretation of sameness between gene therapy products for the purposes of obtaining orphan-drug designation and eligibility for orphan-drug exclusivity. The draft guidance focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease. As laid out in the draft guidance and our regulations, the agencys determination will consider the principal molecular structural features of the gene therapy products, which includes transgenes (the transferred gene) and vectors (the vehicle for delivering the transgene to a cell).

With the large volume of products currently being studied, gene therapy product developers have asked the agency important questions about orphan-drug designation incentives to develop products for rare diseases with very small patient populations. The draft guidance has potential positive implications both for product developers and patients by providing insight into the agencys most current thinking on the sameness of products, and thus, not discourage the development of multiple gene therapy products to treat the same disease or condition. For patients, this policy could help lead to the development and approval of multiple treatments, creating a more competitive market with choices. We encourage stakeholders to provide their comments.

In sum, these policy documents are representative of efforts to help advance product development in the field of gene therapy. We will continue to work with product innovators, sponsors, researchers, patients, and other stakeholders to help make the development and review of these products more efficient, while putting in place the regulatory controls needed to ensure that the resulting therapies are both safe and effective. We also encourage developers of new gene therapy products to make full use of our expedited programs available for products intended to address unmet medical needs in the treatment of serious or life-threatening conditions. These programs include breakthrough therapy designation, regenerative medicine advanced therapy designation, and fast track designation, as well as priority review and accelerated approval. Developers should pursue these programs whenever possible to help bring the benefits of important advances to patients as soon as possible. We believe our work will help advance innovations in a way that assures their safety and effectiveness, provides new therapeutic choices to patients and providers and continues to build confidence in this novel and emerging area of medicine.

The FDA is an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

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FDA Continues Strong Support of Innovation in Development of Gene Therapy Products - FDA.gov

Interpreting Sameness Gene Therapy Products Orphan Drug Regulations – FDA.gov

Docket Number: FDA-2019-D-5392 Issued by:

Guidance Issuing Office

Office of Medical Products and Tobacco, Center for Biologics Evaluation and Research

This guidance provides FDAs current thinking on determining sameness of human gene therapy products under FDAs orphan drug regulations for the purpose of orphan-drug designation and orphan-drug exclusivity. This guidance is intended to assist stakeholders, including industry and academic sponsors who seek orphan-drug designation and orphan-drug exclusivity, in the development of gene therapies for rare diseases. This guidance focuses specifically on factors that FDA generally intends to consider when determining sameness for gene therapy products and does not address sameness determinations for other types of products.

You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))

If unable to submit comments online, please mail written comments to:

Dockets ManagementFood and Drug Administration5630 Fishers Lane, Rm 1061Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2019-D-5392 .

01/30/2020

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Interpreting Sameness Gene Therapy Products Orphan Drug Regulations - FDA.gov

Pfizer lays out gene therapy aspirations – BioPharma Dive

Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine.

The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies.

Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.

Company executives weren't, however, asked to answer how Pfizer views the emerging gene therapy competition. BioMarin Pharmaceutical looks set to get to the market earlier in hemophilia A than Pfizer, while Uniqure in hemophilia B and Sarepta Therapeutics in Duchenne muscular dystrophy appear ahead.

Pfizer's hemophilia A project, the Sangamo Therapeutics-originated SB-525, is up against BioMarin's valrox, which has been submitted to the Food and Drug Administration for an approval decision later this year.

In hemophilia B, fidanacogene elaparvovec, licensed from Roche subsidiary Spark Therapeutics, is in a neck-and-neck race with UniQure's etranacogene dezaparvovec in Phase 3 testing. Duchenne research, meanwhile, is led by Sarepta, which is launching a Phase 3 trial of its drug this year, putting Pfizer's at a disadvantage.

Other than announcing its intent to launch Phase 3 trials in hemophilia A and Duchenne, Pfizer didn't provide much more detail about these clinical programs. Mikael Dolsten, Pfizer's chief scientific officer, said more could be revealed about the DMD program at an upcoming research & development day.

Progress on that project had been delayed after one patient was hospitalized with kidney complications, but Dolsten said trial investigators had dosed additional patients. The Phase 2 will wrap up this spring, and the new data and longer follow-up will help guide a Phase 3 trial design, the company said.

Dolsten also described the hemophilia A project as having a 'best-in-class profile," even though BioMarin's valrox has impressed hematologists with its ability to increase expression of a key blood-clotting protein.

In addition, he said the company hopes it can bring one new gene therapy into its pipeline per year.

Building its drug development portfolio is one reason why the company has chosen not to buy back shares, said CEO Albert Bourla.

He pointed to the company's need in the past to buy back shares to support their valuation because of revenue declines, but now he said the company is in a different strategic position.

"The company is going to have a best-in-class revenue growth story," he said. "We can use the capital to invest in good Phase 2, Phase 3 assets to grow our pipeline."

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Devastation, then hope: Platteville baby first in state treated for rare condition after screening – Madison.com

Dr. Jennifer Kwon, a pediatric neurologist at UW Health, tests Piper Droessler's strength. Born to Caiti and Ben Droessler (background), the Platteville baby was the first child in Wisconsin identified through newborn screening to have spinal muscular atrophy, a potentially fatal muscle-wasting disease. She was also the state's first such baby to receive a gene therapy thought to be a cure.

Like their first two children, Caiti and Ben Droesslers daughter Piper, born in late November, looked healthy, with plump cheeks and tufts of reddish hair.

But a week later, the Droesslers learned Piper, unlike her siblings, had spinal muscular atrophy, a rare muscle-wasting disease. In its most severe form, SMA takes away the ability to walk, talk, swallow and breathe, often becoming fatal by age 2.

Wisconsin began testing newborns for the genetic disorder in October. Piper was the first to test positive, which led to a second milestone: In December, she became the states first child identified through screening to get a new gene therapy for SMA, before developing any sign of weakness.

By counteracting the condition before it can take hold, the treatment is thought to be a cure and should allow her to live a normal life.

The fact that we were able to catch it before theres any loss of function is just amazing, said Caiti Droessler, 32, of Platteville.

Caiti and Ben Droessler, of Platteville, had never heard of spinal muscular atrophy, a potentially fatal muscle-wasting disease, until they learned a week after the Nov. 25 birth of their daughter, Piper, that she tested positive for it. She received a gene therapy that is expected to prevent the condition.

Pipers strength and tone and recoil are all what I would expect, Dr. Jennifer Kwon, a UW Health pediatric neurologist overseeing Pipers care, said in January while examining her during a checkup. The expectation is that she will continue to function normally.

The gene therapy, an intravenous infusion called Zolgensma, was approved by the Food and Drug Administration in May. At $2.1 million, it is believed to be the drug markets most expensive one-time treatment.

Concerns about the cost grew in August, when the FDA said manufacturer Novartis manipulated data involving tests on mice and waited until after approval to report the problem. The FDA said it was investigating, but the findings do not change the agencys positive assessment of the drugs human trials.

The first SMA drug, Spinraza, approved in 2016, costs $750,000 the first year and $375,000 each subsequent year. It is taken for life.

Zolgensmas $2.1 million price is no more ludicrous than paying more than $1,000 a month to rent a portable ventilator, especially when that one treatment will likely eliminate the need for the ventilator and many other costs associated with SMA care, Nathan Yates, a financial consultant who has SMA, wrote for the biomedical news website STAT.

At UW Health, about 40 SMA patients have been treated with Spinraza and 11 have received Zolgensma, including three during clinical trials and three in a special access program prior to approval, Kwon said.

UW Hospital staff draw some of Piper's blood. She was later weaned off steroids after receiving a new gene therapy to prevent SMA.

UW requires insurance approval before giving the drugs. Some insurers initially refused but eventually agreed to pay, Kwon said.

About 1 in 11,000 babies is born with SMA, or fewer than 400 a year nationwide. If both parents are carriers, as the Droesslers learned they are, theres a 25% chance each child will have it.

The condition is caused by a mutation in a gene that normally makes a protein needed by nerve cells that control muscles. Without the protein, the nerve cells die and people lose the ability to move.

Newborn screening

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Spinraza helps a related gene make more of the protein. Zolgensma replaces the mutated gene with a normal one, restoring regular protein production.

Any weakness developed before Zolgensma is given isnt reversed, but the drug is expected to prevent additional problems, Kwon said. If given shortly after birth, before any symptoms develop, the treatment is considered a cure, she said.

However, there is no proof the gene therapy will be long-lasting; the oldest children to receive the drug are 5 years old and doing well. We have the theoretical expectation that they will have normal motor function for their lifetime, Kwon said.

The availability of the treatments has led 17 states to add SMA to their newborn screening programs, according to Cure SMA. Another 15 states have approved doing so.

Wisconsin added SMA to its program Oct. 15. Through blood collected from heel pricks before babies leave the hospital, the state now tests for 47 conditions, many of which can be offset with special diets or medications.

Without family history, newborn screening is the only way we can find (SMA) before symptoms appear, said Dr. Mei Baker, co-director of the newborn screening lab at UW-Madisons Wisconsin State Laboratory of Hygiene.

Four to six new cases of SMA are expected in Wisconsin each year, Baker said.

So far, Piper Droessler remains the only baby to test positive through screening.

Caiti and Ben Droesslers 6-year-old daughter and 3-year-old son dont have SMA, and the couple had never heard of the condition. Caiti works at Southwest Health in Platteville, in human resources. Ben runs a masonry restoration business with his dad and brother.

A week after Piper was born Nov. 25, they got a call that she had SMA.

It was very scary, one of those phone calls you never want to get, Caiti Droessler said.

The next day, they met with Kwon in Madison and decided to try Zolgensma as quickly as possible. Their health insurance, Quartz, approved, and Piper received the hour-long infusion at UnityPoint Health-Meriter on Dec. 18.

The couple is thankful for their fortunate circumstances. If Piper had come six weeks earlier or in a state without SMA screening they would not have known about the disorder in advance.

By the time we found out, she would have already presented with weakness, and at that point, the damage is done, Caiti Droessler said. I feel very blessed that we were given this opportunity to keep her well.

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Devastation, then hope: Platteville baby first in state treated for rare condition after screening - Madison.com

FDA guidance on gene therapies development and manufacturing – BioPharma-Reporter.com

With more than 900 investigational new drug (IND) applications for ongoing clinical studies related to gene therapies, and with the number of advanced therapy medicinal products at clinical stage worldwide exceeding 1,000, the US Food and Drug Administration (FDA) this week released a number of policies.

The policies, addressed to developers and manufacturers, include six final guidance documents on gene therapy manufacturing and clinical development of products, following up to respective draft guidance documents released in 2018, and a draft guidance related to orphan drug designations for therapeutic candidates.

Scientific development in this area is fast-paced, complex, and poses many unique questions during a product review, commented Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, adding The framework we construct for product development and review will set the stage for continued advancement of this cutting-edge field.

Regarding the draft guidanceInterpreting Sameness of Gene Therapy Products Under the Orphan Drug Regulations, the agency explained that it focuses on how the FDA will evaluate differences between gene therapy products when they are intended to treat the same disease.

The final guidance titled Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) aims to inform sponsors on how to provide sufficient CMC information, in order to assure product safety, identity, quality, purity, and strength (including potency) of the investigational product and to be able to claim market authorization from the regulatory body.

Addressed to developers and manufacturers of retroviral vector-based human gene therapy products, the second document titled Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus (RCR) during Product Manufacture and Patient Follow-up determines testing for RCR during manufacture, as well as the regulations for follow-up monitoring of patients who have received such treatments.

Titled Long-Term Follow-Up After Administration of Human Gene Therapy Products, the third document includes recommendations by the FDA regarding the design of long-term follow-up studies for the collection of data on delayed adverse events.

Specifically, the FDA suggests that, as a result of long-term exposure to an investigational gene therapy, patients may be at increased risk of undesirable and unpredictable outcomes, and therefore they may be monitored for an extended period of time past the active follow-up period. The document outlines several factors based on which a risk assessment should be performed to determine the necessity of long-term monitoring for each product.

Another guidance of the FDA is focused on Human Gene Therapy for Hemophilia, and it provides recommendations regarding the clinical trial design for such therapies, as well as addressing discrepancies between Hemophilia A and B coagulation factors activity assays.

Focusing on Human Gene Therapy for Retinal Disorders, the fourth FDA guidance includes recommendations related to product development, preclinical testing, and clinical trial design for such gene therapy products.

Finally, the guidance on Human Gene Therapy for Rare Diseases, with suggestions on the clinical design for such products, is needed, according to the FDA, due to the limited study population size and potential feasibility and safety issues. Moreover, the FDA cites issues related to the interpretability of bioactivity/efficacy outcomes that may be unique to rare diseases or to the nature of the product.

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FDA guidance on gene therapies development and manufacturing - BioPharma-Reporter.com

Global Gene Therapy Industry Outlook 2020-2024 – Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities – P&T Community

DUBLIN, Jan. 30, 2020 /PRNewswire/ -- The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is projected to reach USD 13,005.6 million by 2024 from an estimated USD 3,814.9 million in 2019, at a CAGR of 27.8% during the forecast period.

This report provides a detailed picture of the global gene therapy market. It aims at estimating the size and future growth potential of the market across different segments (by vector, indication, delivery method, and region). The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper the market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast period

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. In 2018, the non-viral vectors segment accounted for the largest share of this market. However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast period

Geographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Key Topics Covered

1 Introduction

2 Research Methodology 2.1 Research Data2.2 Secondary Data2.2.1 Secondary Source2.3 Primary Data2.4 Market Size Estimation2.4.1 Bottom-Up Approach2.4.2 Bottom-Up Approach for Non-Viral Vectors and Viral Vectors Market2.4.3 Growth Forecast2.5 Market Breakdown and Data Triangulation2.6 Assumptions for the Study

3 Executive Summary

4 Premium Insights 4.1 Gene Therapy Market Overview4.2 North America: Market, By Vector (2018)4.3 Geographical Snapshot of the Market

5 Market Overview 5.1 Introduction5.2 Market Dynamics5.2.1 Drivers5.2.1.1 High Incidence of Cancer and Other Target Diseases5.2.1.2 Product Approvals5.2.1.3 Funding for Gene Therapy Research5.2.2 Opportunities5.2.2.1 Strong Product Pipeline5.2.3 Challenges5.2.3.1 High Cost of Treatments

6 Gene Therapy Market, By Vector 6.1 Introduction6.2 Non-Viral Vectors6.2.1 Oligonucleotides6.2.1.1 North America Accounted for the Largest Share of the Oligonucleotides Segment6.2.2 Other Non-Viral Vectors6.3 Viral Vectors6.3.1 Retroviral Vectors6.3.1.1 Gamma-Retroviral Vectors6.3.1.1.1 The Availability of A Wide Range of Gamma-Retroviral Vectors Supports the Growth of This Market6.3.1.2 Lentiviral Vectors6.3.1.2.1 North America Accounted for the Largest Share of the Lentiviral Vectors Segment6.3.2 Adeno-Associated Virus Vectors6.3.2.1 Possible Applications in In Vivo Applications Have Driven Interest in Adeno-Associated Virus Vectors6.3.3 Other Viral Vectors

7 Gene Therapy Market, By Indication 7.1 Introduction7.2 Neurological Diseases7.2.1 Neurological Diseases Account for the Largest Share of the Market7.3 Cancer7.3.1 Cancer is Expected to Show the Highest Growth in This Market7.4 Hepatological Diseases7.4.1 Increasing Prevalence of Hepatitis B Infections Will Support Market Growth7.5 Duchenne Muscular Dystrophy7.5.1 North America Accounted for the Largest Share of the Dmd Gene Therapy Segment7.6 Other Indications

8 Gene Therapy Market, By Delivery Method 8.1 Introduction8.2 In Vivo Gene Therapy8.3 Ex Vivo Gene Therapy

9 Gene Therapy Market, By Region 9.1 Introduction9.2 North America9.2.1 US9.2.1.1 The US Dominates the Global Market9.2.2 Canada9.2.2.1 Growing Burden of Cancer Will Support Market Growth in Canada9.3 Europe9.3.1 Germany9.3.1.1 Germany Accounted for the Largest Share of the Market in Europe9.3.2 France9.3.2.1 Increasing Cancer Incidence Supports Market Growth9.3.3 UK9.3.3.1 Rising Incidence of Melanoma Will Drive Demand in the UK9.3.4 Italy9.3.4.1 High Incidence of Targeted Diseases and Increasing Per Capita Healthcare Spending Will Drive Market Growth in Italy9.3.5 Spain9.3.5.1 Non-Viral Vectors Dominate the Spanish Market, By Vector9.3.6 Rest of Europe9.4 Asia Pacific9.4.1 Japan9.4.1.1 Japan Dominates the APAC Market for Gene Therapy9.4.2 China9.4.2.1 Rising Prevalence of Cancer and Established Base for Gene Therapy are Supportive Factors in China9.4.3 Rest of APAC9.5 Rest of the World

10 Competitive Landscape 10.1 Overview10.2 Market Share Analysis, 201810.3 Key Strategies10.4 Competitive Leadership Mapping (2018)10.4.1 Visionary Leaders10.4.2 Innovators10.4.3 Dynamic Differentiators10.4.4 Emerging Companies

11 Company Profiles 11.1 Biogen11.2 Gilead Sciences, Inc.11.3 Amgen, Inc.11.4 Novartis AG11.5 Orchard Therapeutics PLC11.6 Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)11.7 Molmed S.p.A.11.8 Anges, Inc.11.9 Bluebird Bio, Inc.11.10 Human Stem Cells Institute (HSCI)11.11 SIBIONO Genetech Co. Ltd.11.12 Shanghai Sunway Biotech Co. Ltd.11.13 Uniqure N.V.11.14 Gensight Biologics S.A.11.15 Celgene Corporation (A Bristol-Myers Squibb Company)11.16 Cellectis11.17 Sangamo Therapeutics11.18 Mustang Bio11.19 AGTC (Applied Genetic Technologies Corporation)11.20 Poseida Therapeutics, Inc.

For more information about this report visit https://www.researchandmarkets.com/r/h6pehn

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com

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Global Gene Therapy Industry Outlook 2020-2024 - Strong Product Pipeline Gives Rise to Lucrative Growth Opportunities - P&T Community