Page 11234..1020..»

Category Archives: Cf

Hudson girls stifled by hot CF/M goalie – RiverTowns

Posted: January 18, 2020 at 10:56 am

HUDSON, Wis.-- The Hudson girls hockey team ran into a hot goalie in a 2-1 Big Rivers Conference loss at home to Chippewa Falls/Menomonie Tuesday night, Jan. 14.

The Raiders fired 43 shots at Sabre goalie Haley Frank, including 20 in the second period, but the only one that found the back of the net came off the stick of Leah Parker in the first period.

Parkers unassisted goal 10 minutes, 43 seconds into the game gave the Raiders a 1-0 lead, but the only scoring the rest of the way came from the Sabres. Ella Ausman tied it up at the 12:02 mark of the second period and Sidney Polzin netted the game-winner just over a minute into the third to give Chippewa Falls/Menomonie its first BRC victory of the season.

Hudson goalie Alayna Kunshier finished with 15 saves as the Raiders slipped to 4-9-1 overall, 1-2-0 in conference play.

Hudson had its nonconference game at Onalaska scheduled for Saturday, Jan. 18, cancelled by weather. After visiting the sixth-ranked St. Croix Valley Fusion (11-5-0) in River Falls Tuesday, Jan. 21, the Raiders will host University School of Milwaukee (3-7-4) Friday, Jan. 24, at 7 p.m.

Visit link:

Hudson girls stifled by hot CF/M goalie - RiverTowns

Posted in Cf | Comments Off on Hudson girls stifled by hot CF/M goalie – RiverTowns

Why Blue Jays Would Be OK with Grichuk in CF – Jays From the Couch

Posted: at 10:56 am

The Toronto Blue Jays have a collection of outfielders from which t hey have to select their center fielder. it doesnt look as though theyre looking to pull the trigger on a big deal to land a Starling Marte, so theyll head to Spring Training with their crop of in house options and hope someone takes the job and runs with it.

None of the options the club has is currently what we would call ideal. They guys like Anthony Alford and Jonathan Davis who are more what youd think of when you think of a natural CF. But, they have not been able to show they can handle big league pitching, at least not to the point where they should be given everyday at bats. Lourdes Gurriel Jr.has found a home in left, even if some think it would be worth trying him in CF. Teoscar Hernandez has speed and saw over 600 innings there last season. But, his -7 DRS and UZR/150 of -13.2 look worse than his -1 OAA. In fact, many are ready to limit him to being the DH in 2020.

All of this leads me to be more and more comfortable with Randal Grichuk in CF. Firstly, the guy is good for 25-30 home runs. I mean, he had better be since his 2019 OBP was .280. Maybe Cavan Biggio can work with him on that. Regardless, Grichuk gives you the power and that is difficult to ignore.

The defensive side of things is what really has me thinking that Grichuk would be just fine in CF. In nearly 2000 career innings, he has put up 14 DRS in that position, including 2 last season. His career UZR/150 is -1.2, but -0.6 in 2019. He put up 2 of his 6 OAA in 2019 in CF. Grichuk is a solid defender. Hes not likely to win a Gold Glove any time soon, but he can be relied upon for the bulk of the playing time.

Grichuk is also not going to be Superman out there. Fangraphs tells us that he made zero Impossible or Remote plays, 20% of the Unlikely, but 100% of the Even, 100% of the Likely and 99.1% of the routine plays. Hes not flashy and wont end up on highlight reels, but he is dependable.

None of this is to suggest that Randal Grichuk is someone I would choose in CF if I were building a team. In a perfect world, he would be playing in right field for the Blue Jays. However, in the context of the 2020 season, considering the options available to manager, Charlie Montoyo, it looks like Grichuk will get the nod for the most playing time. Since there is no pressure to win right now, the club can afford to use what they have until they can find a better option.

Maybe moving Biggio to CF makes the overall club better. Doubtful, but maybe. Maybe Gurriel can make the transition look easy. Maybe. Maybe Hernandez will prove to be more than a CF back up. Maybe Alford finds his bat in 2020. Maybe Davis does. Time will tell. Until then, the Blue Jays will go with Randal Grichuk in CF and that is OK with me.

Become a Patron!

Shaun Doyle is a long time Blue Jays fan and writer! He decided to put those things together and create Jays From the Couch. Shaun is the host of Jays From the Couch Radio, which is highly ranked in iTunes, and he has appeared on TV and radio spots.

Originally posted here:

Why Blue Jays Would Be OK with Grichuk in CF - Jays From the Couch

Posted in Cf | Comments Off on Why Blue Jays Would Be OK with Grichuk in CF – Jays From the Couch

Newly approved cystic fibrosis treatment offers promise – Midland Daily News

Posted: November 19, 2019 at 11:49 am

Omar P. Haqqani, for the Daily News

Newly approved cystic fibrosis treatment offers promise

On October 21, the U.S. Food and Drug Administration (FDA) approved a new therapy that could improve the lives of thousands of people living with cystic fibrosis (CF). The first triple combination therapy has been made available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients twelve years and older with cystic fibrosis that have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are approximately 2,000 known mutations of the CFTR gene, but the most common mutation is the F508del mutation. It is estimated to represent 90% of the cystic fibrosis population. Trikafta is very effective on this mutation. The Cystic Fibrosis Foundation (CFF) calls it, "the single greatest therapeutic advancement in the history of CF."

What is cystic fibrosis?

Cystic fibrosis mutations prevent CFTR proteins to become functional and that prevents the movement of chloride to the cell surface in the body. Chloride is an element of salt and when its movement is hindered, mucus in various organs becomes thick and sticky. The improperly developed mucus causes germs and bacteria to remain in the lungs. This results in infection, inflammation and other complications, including respiratory failure.

CF also presents a danger in the pancreas. The mucus causes digestive enzymes not to function properly, resulting in malnutrition and poor growth, due to the prevention of the release of digestive enzymes. Mucus can also cause liver disease by blocking the bile duct. It may also alter the ability for men to father children.

In the U.S., more than 30,000 people are living with cystic fibrosis and more than 70,000 are diagnosed worldwide. More than 50% of them are age 18 or older.

Symptoms of cystic fibrosis

Symptoms of CF appear in proportion to damage to particular organs. The most common and noticeable symptoms occur when the lungs become damaged. Lung infections such as pneumonia and bronchitis, along with wheezing, or shortness of breath are frequent symptoms. Additionally, persistent coughing may occur and often produce phlegm.

When CF affects other organs, symptoms may include salty tasting skin, poor weight gain, despite a good appetite, and infertility. Other symptoms may be bulky stools or problems with bowel movement.

Diagnosis and treatment

CF is a genetic disease. 75% of cases are diagnosed before a child is two years old. As with the symptoms, treatment is focused on the organs exhibiting the most impact from the mutation. In looking at specific symptoms, a physician may prescribe airway clearance, pancreatic enzyme supplements and individualized physical fitness programs. These therapies have often proven effective.

Nasal inhalers that include antibiotics to keep nasal passages clear are used in airway clearance therapy. To improve the absorption of various nutrients, pancreatic enzyme supplement capsules may be prescribed, along with multivitamins.

The available therapies that target the defective protein are treatment options for some patients with cystic fibrosis, but many patients have mutations that are ineligible for treatment. The new therapy is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively.

The prescribing information for Trikafta includes warnings related to elevated liver function tests (transaminases and bilirubin), the use at the same time with other products that are inducers or inhibitors of another liver enzyme called Cytochrome P450 3A4 (CYP3A), and the risk of cataracts. Patients and their caregivers should speak with a health care professional about these risks and any medicines they take before starting treatment.

Patients with cystic fibrosis should also speak with their physicians about having tests performed to understand which gene mutations they have.

To learn more about cystic fibrosis, its symptoms and treatment, log on to vascularhealthclinics.org.

Ask Dr. Haqqani

If you have questions about your cardiovascular health, including heart, blood pressure, stroke lifestyle and other issues, we want to answer them. Please submit your questions to Dr. Haqqani by e-mail at questions@vascularhealthclinics.org.

Dr. Omar P. Haqqani is the chief of Vascular and Endovascular Surgery at Vascular Health Clinics in Midland: http://www.vascularhealthclinics.org

See the article here:

Newly approved cystic fibrosis treatment offers promise - Midland Daily News

Posted in Cf | Comments Off on Newly approved cystic fibrosis treatment offers promise – Midland Daily News

Reg CF Funding Portals: 50 in Total with Several Exits and Several Additions. Is Reg CF Ready to Scale? – Crowdfund Insider

Posted: at 11:49 am

Periodically, Crowdfund Insider revisits the Reg CF sector of online capital formation. Reg CF or Regulation Crowdfunding may have garnered most of the attention from popular media but really there are three individual crowdfunding exemptions including Reg A+ and Reg D 506c.

Under Reg A+ you must file an extensive offering circular with the entire offering process costing around $300,000, according to one estimate. But Reg A+ enables an issuer to raise up to $50 million from both accredited and non-accredited investors.

Under Reg D 506c, you may raise an unlimited amount of money but only from accredited investors. This is the most popular crowdfunding exemption and Reg D (5o6c and 5o06b) is a trillion-dollar market.

Issuers using Reg CF may only raise $1.07 million and must utilize a FINRA regulated Funding Portal or a broker-dealer. Due to the low cap on funding, frequently issuers will do a side-by-side Reg D 506c offering to circumvent the extremely low amount you may raise.

Last time CI revisited the number of approved Funding Portals was in July. Since that time, several new funding portals have joined the approved list and several have exited.

Regarding Reg CF funding portal exits two more have departed this sector of crowdfunding.

EquityBender based in Charleston, South Carolina, is no more. The domain just indicates a private site.

Seeding VR is the other exit. As the name indicates, Seeding VR was targeting the virtual reality sector. Apparently, it even attempted to launch a crowdfunding platform in the UK. Today, both domains simply time out.

This brings the total of funding portal exits to 12 with at least two the direct result of some time of enforcement action: UFP LLC and DreamFunding Marketplace.

The three additions to the list bring the total to 50 FINRA approved funding portals with one in question as Fundpaas has long been on the suspended list and is expected to join the exits.

The three recent additions include:

Fundopolis has yet to list its first offering but appears ready to launch its first issuer. According to its website, Fundopolis expects to also enable issuers to raise capital under both Reg A+ and Reg D as well.

Infrashares is described as follows:

InfraShares is a crowdfunding platform that pools investment from individuals into large sums of development capital for critical infrastructure projects (roads, bridges, airports, mass transit, water systems, renewable energy and schools).

This platform is utilizing Reg D as well. Currently, there are two issuers posted on the site -both under Reg D 506c.

Prospect Equity does not appear to have a live site as of yet.

As Crowdfund Insider reported in October, Reg CF has raised over $300 million in securities offerings since the exemption became actionable in May of 2016, providing capital to over 2000 campaigns. This is according to a report by Crowdfund Capital Advisors.

Overall, Reg CF can be called a success as it has helped smaller companies raise much-needed growth capital while creating new jobs. But multiple shortcomings hobble the exemption thus undermining its potential success.

First, the fund cap is widely recognized as far too low.

Average seed rounds in the US stand at about $2.2 million typically using Reg D. A good number raise much more.

In the UK, the most robust crowdfunding market in the world, issuers may raise as much money as they want. A prospectus requirement at 8 million creates a virtual speedbump for issuers looking to raise more than that amount.

The most successful Reg CF Funding Portal, Wefunder, stated earlier this year that limitations to the exemption may make the exemption an option of last resort.

Republic, another leader in the Reg CF sector, published a letter providing the perspective from issuers that have utilized Reg CF to raise growth capital. The letter, signed by 23 different founders and CEOs of early-stage firms, indicated that Reg CF has its benefits but suffers from serious limitations.

Every industry participant has voiced their concerns to both Congress and the Securities and Exchange Commission (SEC). While some policymakers have supported common-sense updates, since 2016, little has been accomplished.

And it is not just the funding cap that undermines Reg CF.

A report by the SEC from this past summer touched upon many of the issues.

Common sense changes that allow for special purpose vehicles (SPVs) that safeguard smaller investors while making it simpler to gain access to higher-quality deals is a no brainer.

There is also the 12g trap that may compel a company to become a reporting company once it has 500 investors. A problem that is antithetical to investment crowdfunding.

The Association of Online Investment Platforms (AOIP), an advocacy group for online capital formation, has posted a position paper with its goals.

So is Reg CF ready to scale? Probably not without some changes to the rules. Many of the larger platforms now have broker dealer licenses and offer other services. Doing enough $1 million deals in a year to cover your costs can be quite difficult.

The SEC is currently going through a regulatory review, as defined by a concept release, which seeks to improve the exemption ecosystem while harmonizing the alphabet soup of rules. This may be the best opportunity the industry has to see some impactful improvements. If not, it will be up to Congress to step up and do the job. Dont hold your breath.

Read more from the original source:

Reg CF Funding Portals: 50 in Total with Several Exits and Several Additions. Is Reg CF Ready to Scale? - Crowdfund Insider

Posted in Cf | Comments Off on Reg CF Funding Portals: 50 in Total with Several Exits and Several Additions. Is Reg CF Ready to Scale? – Crowdfund Insider

The ‘CF World’: Fighters work toward a cure – Post Register

Posted: November 17, 2019 at 2:22 pm

BLACKFOOT Bingham County has shown itself as a community that rallies behind special causes and special needs whenever they arise.

The fight against cystic fibrosis (CF) is one of those causes. Its a a hereditary disease that affects the lungs and digestive system, with the body producing thick, sticky mucus that can clog the lungs and obstruct the pancreas. CF can be life-threatening, and people with the condition tend to have a shorter-than-normal life span.

Its a disease that has reached around the county. Its a disease that brings a rallying cry every July with the Wolverine Canyon Marathon, which raises awareness of CF and helps raise money for research into ultimately finding a cure, highlighting one person each year in the county who battles CF.

This year, with help from donors and local participants including sponsors and the runners themselves, the marathon raised $14,863.44. The VanOrden family in the Thomas area received a check for $7,431.42. They plan to donate a portion of of that to cystic fibrosis-related causes, use some of the funds to pay off medical expenses for their 4-year-old son, Taylor, who has CF, and put the remaining money in a medical fund for Taylor to use for future medical expenses.

In 2017, Frannie Baumgartner Crumley of Blackfoot was highlighted, turning 40 that year, having a double lung transplant to extend her life. Emersyn Drollinger, a young Shelley girl, was highlighted in 2018. She was diagnosed with cystic fibrosis as an infant. Taylor VanOrden started showing signs of the disease just a couple of days after being born at Eastern Idaho Regional Medical Center.

Frannie daughter of Alice and Gary Baumgartner lost a sister, Julia, to the disease.

The disease and the daily treatment that goes with it is all that Taylor has ever known, aside from the love and support of his family and friends. Hes become so accustomed to the daily ritual, months short of his fifth birthday, that there are things he does on his own to help himself, such as strapping on his own percussion vest which is needed at least twice a day, used to relieve the mucus that can build up in the body and become thick and sludgy much like overused motor oil in a cars engine, with life-threatening results if left untreated.

CF AWARENESS

We were in ignorant bliss for the first 36 hours after Taylor was born, his father Jeff says.

A couple of days after he was born, Jeff and Taylors mother, Lyndsey, knew there were complications.

That was when he threw up greenish mucus. He hadnt had a bowel movement in 36 hours, finding blockage in the small intestine. On day two after being born, Taylor was Life Flighted to Primary Childrens Medical Center in Salt Lake City.

At the time, doctors were unsure whether it was CF or an intestinal disorder. Then came the family history. Neither parent had known there had been CF in the family until Lyndsey was told that her father had a cousin who died of CF at age 18, an average lifespan for people with CF back then.

There are no outward symptoms at all. Its known as an invisible disease, and its a disease he will likely fight his entire life. But there is still hope that positive steps in funding and research will improve quality of life if not upholding the mantra of the Cystic Fibrosis Foundation (https://www.cff.org/): We will not rest until we find a cure.

Its a mantra the VanOrden family lives with every day for Taylor.

According to the CFF, in people with the disease, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, its unable to help move chloride a component of salt to the cell surface. Without the chloride to attract water to the cell surface, the mucus in various organs becomes thick and sticky.

In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. Minimizing contact with germs is a top concern for people with CF.

In the VanOrden home, having two school-age children in Taylors sister Emma, 9, and brother Tommy, 7, who are exposed to germs on a daily basis means extra caution on a daily basis is required, along with avoiding germs from anywhere. Emma and Tommy have both been tested for CF and are clear.

According to the CFF, signs of CF can include very salty-tasting skin; persistent coughing, at times with phlegm; frequent lung infections including pneumonia or bronchitis; wheezing or shortness of breath; poor growth or weight gain in spite of a good appetite; frequent greasy, bulky stools or difficulty with bowel movements; and male infertility.

A CLOSE-KNIT UNIT

Frannie Baumgartner Crumley calls younger CF fighters {span}Cysters and fiBros, Shes hoped for a cure to be found, so that one day the fighters will be {span}able to take one pill and be cured of CF.{/span}{/span}

{span}{span}Frannie stands as an inspiration to families of those dealing with CF, and there is a tight bond between those families. Its there for support, to keep up the fight and the drive.{/span}{/span}

{span}{span}Theres quite a few families around here who have it, said Lyndsey, who Jeff says is highly involved in a CF advisory board that meets every week. The support theyve seen with the marathon has been a very humbling experience, they said, seeing the community rally together.{/span}{/span}

{span}{span}Frannie is incredible. Shes lived into her 40s, Lyndsey added. Frannie reminds me of what Taylor will be. She has been a role model. Shes given us hope.{/span}{/span}

The goal that Frannies had of getting down to a one-pill cure sounds much better than the 13 maintenance medications Taylor must take daily now, with some of them being adult-sized tablets. One of those medications, Orkambi an oral granule costs around $300,000 per year. Taylor has to take that twice a day, and its delivered in a box keeping them supplied four weeks at a time.

With the medical coverage the family is lucky to have, the family has a copay of $45 a month for Orkambi.

It does cause anxiety, Jeff said.

The VanOrdens say they have a good medical plan, but premiums keep rising substantially. They meet their yearly deductible by the end of January. The government provides no funding for research, which leaves donations through the foundation and efforts such as the Wolverine Canyon Marathon.

The foundation is pushing hard for a cure, Lyndsey said. Its making substantial progress.

Theres something about the CF world. There are a lot of passionate people.

Follow this link:

The 'CF World': Fighters work toward a cure - Post Register

Posted in Cf | Comments Off on The ‘CF World’: Fighters work toward a cure – Post Register

Chicago Fire Receives $75000 in General Allocation Money from Inter Miami CF – Chicago Fire

Posted: at 2:22 pm

CHICAGO (Nov. 14, 2019) The Chicago Fire today announced that it has received $75,000 in 2020 General Allocation Money (GAM) from Inter Miami CF. In exchange, Chicago traded defender Grant Lillard to Inter Miami.

Going back to his days as an Academy player, Grant always represented the Club with pride, said Chicago Fire President and General Manager Nelson Rodrguez. We appreciate what he has given the Club and hope that this opportunity further advances his career.

Lillard, 23, was signed by the Fire on January 18, 2018. Over the last two seasons, the Hinsdale, Ill., native represented the Club on 14 occasions across all competitions, including MLS regular season matches and Lamar Hunt U.S. Open Cup contests.

In 2020, the Fire will return to Soldier Field for its 23rd MLS campaign. Fans can secure a seat for the Clubs 2020 MLS Home Opener on March 21 against Atlanta United FC, or any game of the regular season, with a refundable $20 ticket deposit. Season tickets for the 2020 season are also currently on sale, with packages starting at $25 per game. More information is available at http://www.chicago-fire.com/tickets.

Transaction: Chicago Fire Soccer Club acquires $75,000 in 2020 General Allocation Money from Inter Miami CFin exchange for defender Grant Lillard on Nov. 14, 2019.

Continue reading here:

Chicago Fire Receives $75000 in General Allocation Money from Inter Miami CF - Chicago Fire

Posted in Cf | Comments Off on Chicago Fire Receives $75000 in General Allocation Money from Inter Miami CF – Chicago Fire

Vertex Confirms Northern Ireland Offer Accepted for Cystic Fibrosis Medicines – Business Wire

Posted: at 2:22 pm

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that the Regional Pharmaceutical Procurement Service in Northern Ireland has accepted an offer for access to all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

This means that once the contract is finalized, patients with CF in Northern Ireland ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene will have access to ORKAMBI (lumacaftor/ivacaftor) and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations will have access to SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor. We will support the arrangements being put in place to ensure clinicians will be able to prescribe to eligible patients within the next few weeks.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include people ages 18 years and older who have the R117H mutation and those patients ages 12 months and older who have one of the nine licensed gating mutations.

We are pleased that the nearly 280 eligible cystic fibrosis patients in Northern Ireland will soon have access to CFTR modulators to treat the underlying cause of their disease and we thank the authorities in Northern Ireland for their collaboration and commitment in this agreement, said Ludovic Fenaux, Senior Vice President, Vertex International.

About CF in the UKOver 10,000 people in the UK have CF the second highest number in the world. Nearly 480 people in Northern Ireland have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours, involving nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutationIn people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftorSome mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor)KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements by Mr. Fenaux in the fourth paragraph of this press release and statements regarding our expectations for the patient populations that will be able to access Vertexs medicines and the timing of such access. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

See original here:

Vertex Confirms Northern Ireland Offer Accepted for Cystic Fibrosis Medicines - Business Wire

Posted in Cf | Comments Off on Vertex Confirms Northern Ireland Offer Accepted for Cystic Fibrosis Medicines – Business Wire

Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo – Irish Examiner

Posted: at 2:22 pm

Groundbreaking research has found that the majority of people with cystic fibrosis can be treated successfully.

Researchers at Queens University in Belfast and at the Cystic Fibrosis Unit at St Vincents Hospital Dublin, have identified how a combination of three drugs, known as triple therapy, can tackle the underlying cause of the incurable disease.

The drug, Trikaftatm, targets the root cause of cystic fibrosis (CF), a genetic condition that clogs up the lungs and digestive system, making breathing difficult and often resulting in an early death for those affected.

CF is an inherited chronic disease that primarily affects the lungs and digestive system of about 1,300 children and adults in Ireland and 70,000 worldwide.

That figure, according to Philip Watt of Cystic Fibrosis Ireland, is set to increase by 75% in adults and 25% in children in this country according to ongoing studies by 2025.

This new drug Trikaftatm will benefit 90% of sufferers worldwide, lung function is expected to increase dramatically and will result in a 60% decrease in hospitalisation. This is a hugely welcome advancement. Many of the trials by the Queens University researchers were carried out at several centres in Ireland including St Vincents Hospital Dublin.

Dr Francis Collins, an American researcher who discovered the CF gene in 1989 has already said this is the breakthrough in research he has been waiting for. Many of the trials prior to this did not achieve as much as this study has.

Dr Collins who is the director of the National Institutes of Health writing in The New England Journal of Medicine and the highly respected Lancet medical magazine over the weekend, said these findings indicate that it may soon be possible to offer safe and effective molecularly targeted therapies to 90% of persons with cystic fibrosis.

He said that this should be a cause for major celebration and the best day ever for all of us traveling down this long road together will be the day when the more than 70,000 persons with cystic fibrosis worldwide do not need to take drug therapy at all and there finally is a permanent cure for cystic fibrosis that works for everyone.

Mr Watt added: The Food and Drugs Administration in the US has approved this drug five months ahead of time and now the European Medicines Agency is reviewing it. As a result we would expect this drug to be on the market here by the Summer of next year to those over 12-years-of-age. We would be hopeful that will include children aged as young as two in the future.

The numbers of adults and children increasing in Western Europe is down to advancements in related drugs and quality of services.

Talks and negotiations with the Health Service Executive on this new announcement will, Im sure, commence as a pipeline deal on future drug advancements was agreed with them in 2017 when Orkambi was finally approved. So far we are not privy to the potential costs of this new drug.

However, it is estimated that the drug may cost around $311,000 or 279,000 annually by Vertex, a pharmaceutical company that produces other CF drugs.

Ireland has the highest incidence of CF in the world with one in 19 Irish people being said to carry one copy of the altered gene, with three times the rate of the United States and the rest of the EU.

The organisation has pointed out that it is important to have a network of centres of expertise in dealing with disease.

A defective gene and its protein product cause the body to produce unusually thick, sticky mucus that clogs the lungs and leads to life-threatening lung infections; and obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food.

Researchers believe it is possible to help control the symptoms and delay complications to make the condition easier to live with.

Dr Damian Downey, is a Clinical Senior Lecturer at the Wellcome-Wolfson Institute for Experimental Medicine, Queens University Belfast and co-author on the worldwide trial, set-up to assess the safety and efficacy of a new triple-drug combination called Trikaftatm in patients with CF aged 12 years and older.

The study involved a four-week, randomised, active-controlled trial in 107 patients who had two copies of the (F508del) most common gene mutation.

Dr Downey said: The trial was a success in demonstrating that this drug combination can potentially treat up to 90% of people with CF by addressing the underlying cause of their disease.

This new triple therapy has the potential to transform the lives of people with CF. It results in a significant improvement in lung function and quality of life and also reduces the frequency of chest infections. This treatment will likely alter the future of CF care.

See the original post here:

Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo - Irish Examiner

Posted in Cf | Comments Off on Research finds majority of people with cystic fibrosis can be treated successfully with three-drug combo – Irish Examiner

Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines – Business Wire

Posted: at 2:22 pm

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that NHS Wales has accepted an offer for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England.

This means that once the contract is finalized, patients with CF in Wales ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can access ORKAMBI (lumacaftor/ivacaftor) and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can access SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftor in the coming weeks.

The agreement also offers expanded access to KALYDECO (ivacaftor) to include those patients ages 12 months and older who have one of the nine licensed gating mutations.

Todays announcement is good news for the approximately 270 eligible cystic fibrosis patients in Wales who will soon have access to CFTR modulators to treat the underlying cause of their disease, said Ludovic Fenaux, Senior Vice President, Vertex International. We thank the authorities in Wales for their collaboration in accepting this offer under the same terms as were recently announced in England.

About CF in the UKOver 10,000 people in the UK have CF the second highest number in the world. Over 430 people in Wales have CF. CF is a debilitating, life-shortening inherited condition that causes progressive damage to organs across the body from birth. Currently, there is no cure for CF and half of people in the UK with CF die before they are 32. The daily impact of treatment is significant. It can take up to four or more hours, involving nebulizers, physiotherapy and up to 70 tablets a day. CF accounts for 9,500 hospital admissions and over 100,000 hospital bed days a year. A third of these are used by children under 15.

About ORKAMBI (lumacaftor/ivacaftor) and the F508del mutationIn people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.

Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About SYMKEVI (tezacaftor/ivacaftor) in combination with ivacaftorSome mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.

SYMKEVI is indicated for people with CF ages 12 and older who either have two copies of the F508del mutation or one copy of the F508del mutation and have one of the following 14 mutations in which the CFTR protein shows residual function: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3AG, S945L, S977F, R1070W, D1152H, 2789+5GA, 3272-26AG, or 3849+10kbCT.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About KALYDECO (ivacaftor)KALYDECO (ivacaftor) is the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to keep CFTR proteins at the cell surface open longer to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is indicated in people ages 12 months and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. KALYDECO is also indicated for the treatment of patients with CF ages 18 years and older who have an R117H mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on http://www.ema.europa.eu.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has four approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements by Mr. Fenaux in the fourth paragraph of this press release, statements regarding our expectations for the patient populations that will be able to access Vertexs medicines and the timing of such access, and statements about our expectations regarding a formal agreement in Northern Ireland. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Originally posted here:

Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines - Business Wire

Posted in Cf | Comments Off on Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines – Business Wire

Rare Disease Film Festival Highlights Patient and Researcher Unity in CF, Other Disorders – Cystic Fibrosis News Today

Posted: at 2:22 pm

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare diseases that have been discovered; on this weekend, nearly 60 were shown.

Threading the theme of rare disease, the films on offer traversed topics of treatment hopes and progress, isolation, mental health, and the need for boosted awareness. Films ran between a single minute and 85, some through independent creation and others by corporate or nonprofit sponsorship, by both amateur directors and award-winning documentarians. Genres included fantasy, documentary, animation, silent film, and foreign language.

The festival was co-created by Bo Bigelow, the father of a daughter with a USP7-related disease, and Daniel DeFabio, whose son has Menkes syndrome. It is the second event of its kind the first was held in Boston in October 2017.

This year, the films were hosted at the University of California, San Francisco (UCSF) Mission Bay Conference Center. Fittingly, UCSF directs the Eli and Edythe Broad Center for Regeneration Medicine and Stem Cell Research, one of the largest centers of its kind researching cures for diseases like those featured at the festival.

Many of the films celebrated the rare disease communitys progress from an age of searching for targets in disease origin, to a new age of nailing those discovered targets with innovative treatments. These films included not only the narratives of patients and their caregivers, but also their union with the researchers chasing diagnoses and cures.

The first film shown at the festival, director Andrew Pucios Counting Every Second, chronicles giant axonal neuropathy patient Hannah Samess journey. It follows her crying as she shares her dreams for a future, to receiving a life-altering gene therapy, to coming face-to-face with researchers to thank them for halting her disease.

The Race, a documentary by Dina Rudick, interwove the narratives of a woman named Jenne Coler-Dark with Huntingtons disease and scientist Logan Bishop-Currey, whos hunting a cure. In the film, she steps out of the lab to meet Jennes family.

Ive never met a person with Huntingtons. Ive never seen a face, she said before meeting them. When she did, though, it made her work personal. I now will see Jenne and her kids when I look at the data.

The film festival embodies this theme by aiming to put the often abstract and dire clinical information into the context of real people living their lives. By doing this, Bigelow and DeFabio hope to raise the stakes for researchers, and stick rare diseases at the forefront of the publics mind.

The films also provide a medium for depicting disease on the patients terms. Cystic fibrosis (CF) patient Amanda Korst, who was featured in fianc and director Joe Bergs A Day in My Life, spoke about finding balance in not revealing the most humiliating parts of her disease in the film while also depicting its reality.

You Google [CF] its not great stuff. Dont Google my disease. Its all just bad, bad, bad there. I want to show you my disease myself.

Between showings, filmmakers and people from the various disease communities shared the stage for open forums. In both their films and in the on-stage discussion, they emphasized the importance of realizing that, combined, the rare disease communities form a large family that is anything but disorderly.

When we join with other rare disease communities, we are huge and we have a voice, said Sharon King, the mother of Taylor King, who passed from Batten disease on Sept. 26, 2018.

Films showed the great strides by patients and their family members to raise funds and awareness through activities such as mountain climbing, the formation of nonprofits, and by becoming researchers.

Laura King Edwards, sister to Taylor and a Batten Disease News columnist, has been running half-marathons all across America while blindfolded to support Taylors Tale, a nonprofit that aims to conquer the fatal brain disease. Taylors Tale, which Edwards co-created, is now also dedicated to helping all other rare disease communities via public policy advocacy.

The only thing that will slow us down is if Battens disease is cured, Edwards said. Even then, the rare disease family is our family.

Bigelow and DeFabio plan to continue their rare disease community film rallies in future years. For more information, visit the Disorder: The Rare Disease Film Festival website.

Brad Dell is a deaf 26-year-old with cystic fibrosis. Originally from Hawaii, he received a double-lung transplant from University of California at San Francisco in January 2017. When not traveling, drinking coffee, or reading comics, hes working as the senior director of columns at BioNews Services. (OK, hes still drinking coffee while he works.)

Total Posts: 336

Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

Read the original here:

Rare Disease Film Festival Highlights Patient and Researcher Unity in CF, Other Disorders - Cystic Fibrosis News Today

Posted in Cf | Comments Off on Rare Disease Film Festival Highlights Patient and Researcher Unity in CF, Other Disorders – Cystic Fibrosis News Today

Page 11234..1020..»