Establishment Republicans have been eager to get past the part of the election cycle where Ron Paul has played an outsized role. Rand Paul?s recent endorsement of Mitt Romney divided libertarians, but the Paul heir?s apparent capitulation to business as usual actually underscores how the GOP faces a more complex challenge to the ideological status ...
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From Ron to Rand, the GOP's Paul Problem Isn't Going Away. It Also Isn't a Problem
By AARON LeCLAIR/lbedit7@laramieboomerang.com Tuesday, June 19, 2012
Libertarian Richard Brubaker said he would advocate for the gradual reduction of the federal government and create jobs for the middle class if he is elected to the U.S. House of Representatives this fall.
Brubaker, 59, a truck driver from Riverton, is challenging incumbent Congresswoman Cynthia Lummis for Wyomings lone seat in the House.
The Libertarian concept is something we need to start looking at, he said. We have to stop thinking with our hearts and start thinking with our heads. The heart is not a very good thinking organ.
As the Wyoming Libertarian Partys sole candidate, Brubaker will face Lummis and Democrat Chris Henrichsen of Casper in the general election on Nov. 6.
Brubaker said one of the reasons he chose to run for the House is to work on the gradual reduction of the federal government. He admits that reducing the size of the federal government will not happen overnight.
Its a direction versus something that needs to instantaneously take place, he said. Its going to be up to the next generation, maybe the ones not even voting yet, to figure out all these solutions. I just want to get it started in a direction that we do find solutions.
One way to begin reducing the size of the federal government is to start with high-end government officials, Brubaker said.
We need to start looking at cutting on the top end far more than on the bottom and leave the middle alone, he said. The working class people are already stressed and stretched to the limit.
Another way to reduce the size of the federal government is the budget. If he is elected to the House, Brubaker said he would vote for a federal budget that is no more than 45 percent of estimated revenues.
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Libertarian Party presidential nominee Gary Johnson, left, is given a T-shirt by Groundswell Cannabis Boutique co-owner Don Novak on Monday. (Stephen Mitchell, The Denver Post)
Presidential hopeful Gary Johnson's message to voters is that he's better on civil-libertarian issues than Democrat Barack Obama and better on dollars-and-cents issues than Republican Mitt Romney.
In the West, where several states will be crucial in determining who wins in November, Johnson, running on the Libertarian ticket, is an attractive conveyer of that message.
The erstwhile Republican served two terms as governor of New Mexico, a state where he's polling at 12 percent.
He's a strong advocate of state's rights.
And as an added edge in Colorado, he has raced eight times in the Leadville Trail 100 mountain-bike race and has a long string of extreme-athlete bona fides.
Johnson's strengths introduce an interesting dynamic: If the presidential race in Colorado is as close as some pundits predict, Johnson could end up being compared with third-party candidates Ralph Nader in 2000 and Ross Perot in 1992, viewed as spoilers in those presidential races.
Which party would likely benefit from a Johnson surge?
During a campaign swing through Colorado where he marched in the gay-rights PrideFest on Sunday and spoke in favor of a ballot measure to regulate marijuana like alcohol Monday Johnson downplayed such speculation.
"I've never viewed myself as a spoiler because I believe I'm going to take equally from both sides," Johnson said just before a news conference at a Denver medical-marijuana business.
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Presidential hopeful Gary Johnson, Libertarian from New Mexico, attempts to woo voters
Solomon Islands striker Benjamin Totori made such an impression on Ricki Herbert at the recent OFC Nations Cup that the coach has signed him for the Wellington Phoenix.Totori tormented the All Whites' defence, scoring superb goals...
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Even with the tourists and locals on the islands, the animals still have relatively little fear of humans.
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Carrie Niland: Inside The Galapagos Islands Menagerie (PHOTOS)
18-06-2012 10:53 On Sunday morning's Q&A, Christopher Buser from the VA Maryland Health Care System discusses the care that returning vets need, how they can enroll to get their benefits and how they can prepare to pay for bills they can't afford. To enroll for benefits, vets can visit or call 877-222-VETS.
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18-06-2012 14:35 (Highlights) HHS Secretary Sebelius gathers with women in Madison, Wisconsin, to hear their stories as the economic decision-makers for their families and discuss changes in health care made available by the Affordable Care Act. Learn more about Women and the Affordable Care Act: Covered Preventive Services for Women: Sign up to receive email updates from HealthCare.gov: -- Take health care into your own hands. US Department of Health and Human Services (HHS) http We accept comments in the spirit of our comment policy: HHS Privacy Policy
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Women's Health Living Room Discussions (Madison, WI) - Video
18-06-2012 14:53
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2012 Community Benefit: Exceptional Patient Care - Carolinas HealthCare System - Video
stethoscope and pen resting on a sheet of medical lab test results, with patient file and x-ray or mri film
By TOM MURPHY, Associated Press
One of the biggest misconceptions about President Obama's health care overhaul isn't who the law will cover, but rather who it won't.
If it survives Supreme court scrutiny, the landmark overhaul will expand coverage to about 30 million uninsured people, according to government figures. But an estimated 26 million U.S. residents will remain without coverage a population that's roughly the size of Texas and includes illegal immigrants and those who can't afford to pay out-of-pocket for health insurance.
"Many people think that this health care law is going to cover everyone, and it's not," says Nicole Lamoureux, executive director of the Alexandria, Va.-based National Association of Free & Charitable Clinics, which represents about 1,200 clinics nationally.
[Check it Out: U.S. News & World Report Top Health Insurance Companies.]
To be sure, it's estimated that the Affordable Care Act would greatly increase the number of insured Americans. The law has a provision that requires most Americans to be insured or face a tax penalty. It also calls for an expansion of Medicaid, a government-funded program that covers the health care costs of low-income and disabled Americans. Additionally, starting in 2014, there will be tax credits to help middle-class Americans buy coverage.
The Supreme Court is expected to hand down a decision this month on whether to uphold the law completely or strike down parts or all of it. If it survives, about 93 percent of all non-elderly, legal U.S. residents will be covered by 2016. That's up from 82 percent this year.
Still, millions of illegal immigrants won't qualify for coverage. This population will account for roughly 26 percent of those who will remain uninsured, according to Urban Institute, a nonpartisan think tank.
And many legal U.S. residents will go without insurance, too. About 36 percent of the population that remains uninsured will qualify for Medicaid but won't sign up for various reasons. Others likely will make too much money to qualify for assistance but be unable to afford coverage.
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One of the biggest misconceptions about President Obama's health care overhaul isn't who the law will cover, but rather who it won't.
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Got a cold, a weight problem or pain that wont go away? Theres an app for that.
The surge in smartphone use is spilling over into the mobile health market with consumers using apps to maintain their health by using apps to do everything from check their blood pressure, log calorie intake and even monitor sugar levels. The trend has caught on, and doctors and hospitals are increasingly embracing mobile apps as an effective way to treat certain conditions.
Its a huge market. Its gone from zero before the iPhone and other mobile devices launched to around a $700 million market, says Chris Wasden, PwCs global healthcare innovation leader. Now hospitals are setting up their own app stores and youve got the [National Health Service] in the U.K. recommending doctors prescribe apps rather than have an office visit.
According to a recent survey and study of the mobile health market (mHealth) for PwC Global Healthcare by the Economist Intelligence Unit, among consumers who use mHealth services, 59% report the services have replaced some doctor office visits.
Survey respondents ranked more convenient access to their health-care provider, the reduction of out-of-pocket health-care costs and having greater control over their health as the top three reasons to use mHealth.
Visit one of the mobile app stores and chances are you will quickly stumble on a mHealth app. According to Wasden, there are 12,000 mhealth apps on the market and number that is only expected to grow.
Some studies have shown apps to be more effective than more traditional treatments. According to a study commissioned by PwC, a clinical trial of the WellDoc diabetes management system has shown users of a diabetes management app see their sugar level decrease over a point and a half. By comparison, the FDA considers a new drug that is able to reduce sugar level by half a point as clinically significant.
Diabetes wont be solved with a pill. Its a behavior problem, says Wasden. If you have a tool that transforms a patients behavior they will be more compliant regarding what they eat and what type of activity they do. According to Wasden, 75% of all health-care costs are associated with chronic diseases like diabetes and obesity. If people change their behavior it eliminates the cost, he says.
Mhealth apps go beyond managing diseases. There are medical devices, including an EKG, that connect to your smartphone to check vitals and lenses that can detect skin cancer.
While medical apps are booming in popularity, their potential in the diagnosis and treatment processes are still just taking off. Experts say that in the future patients will be to talk to other apps and sensors will collect data from a user that can be sent to a doctor to monitor a condition. Even car makers are getting into the game: Wasden says some automobile companies are looking at how to use cars as health pods that passively collects biometric information like heart rate and blood-sugar level.
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Health Care is Going Mobile, Consumers Ready, Doctors Sill Leery
One of the biggest misconceptions about President Obama's health care overhaul isn't who the law will cover, but rather who it won't. If it survives Supreme court scrutiny, the landmark overhaul will expand coverage to about 30 million uninsured people, according to government figures.
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Health care overhaul's plan to expand coverage leaves millions behind
WASHINGTON--(BUSINESS WIRE)--
A new report released today by the Department of Health and Human Services (HHS) shows that 3.1 million young adults have gained health insurance because of the health care law. Without the health care law the Affordable Care Act these 3.1 million young adults would not have health insurance. As a result of the law, the proportion of insured adults ages 19 through 25 has increased to nearly 75 percent.
The Affordable Care Act requires insurers to allow young adults to remain on their parents family plans until their 26th birthday, even if they move away from home or graduate from school. This policy took effect on September 23, 2010.
Today, because of the health care law, more than 3 million more young adults have health insurance, said HHS Secretary Kathleen Sebelius. This policy doesnt just give young adults and their families peace of mind, it also gives them freedom. It means that as they begin their careers, they will be free to make choices based on what they want to do, not on where they can get health insurance.
Before the Affordable Care Act, young adults were the age group least likely to have health insurance. Not only were young adults more likely to be uninsured, they were also more than twice as likely as older adults to lose private insurance coverage once they had it. Some young adults lost coverage when they became too old to qualify as a dependent on their parents plans, and others lost coverage as they graduated from school or changed jobs.
A similar report released in December 2011 showed that 2.5 million young adults who would otherwise have been uninsured had gained coverage through June 2011. Using the most recent information on insurance coverage from the National Health Interview Survey conducted by the National Center for Health Statistics, todays report finds that from September 2010 to December 2011 the percentage of adults ages 19 through 25 with insurance coverage increased from 64.4 percent to 74.8 percent. That translates to 3.1 million young adults with coverage. This increase continues the steady upward trend in insurance coverage among young adults since the Affordable Care Act went into effect.
Starting in 2014, there will be even more health coverage options available to young adults when Affordable Insurance Exchanges, premium tax credits, and the Medicaid expansion go into effect.
To see the new HHS report, please visit: http://aspe.hhs.gov/aspe/gaininginsurance/rb.shtml
To see the National Center for Health Statistics Report, please visit: http://www.cdc.gov/nchs/
To learn more about young adults, like Abby Schanfield, who are helped by the under 26 provision, please visit: http://www.healthcare.gov/blog/2012/04/mycare_abby.html
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New health care law helps more than 3 million young adults get and keep health coverage
One of the biggest misconceptions about President Obama's health care overhaul isn't who the law will cover, but rather who it won't.
If it survives Supreme Court scrutiny, the landmark overhaul will expand coverage to about 30 million uninsured people, according to government figures. But an estimated 26 million Americans will remain without coverage -- a population roughly the size of Texas and includes illegal immigrants and those who can't afford to pay out-of-pocket for health insurance.
"Many people think that this health care law is going to cover everyone, and it's not," says Nicole Lamoureux, executive director of the Alexandria, Va.-based National Association of Free and Charitable Clinics, which represents about 1,200 clinics nationally.
To be sure, it's estimated that the Affordable Care Act would greatly increase the number of insured Americans. The law has a provision that requires most Americans to be insured or face a tax penalty. It also calls for an expansion of Medicaid, a government-funded program that covers the health care costs of low-income and disabled Americans. Additionally, starting in 2014, there will be tax credits to help middle-class Americans buy coverage.
The Supreme Court is expected to hand down a decision this month on whether to uphold the law completely or strike down parts or all of it. If it survives, about 93 percent of all non-elderly, legal U.S. residents will be covered by 2016. That's up from 82 percent this
Still, millions of illegal immigrants won't qualify for coverage. This population will account for roughly 26 percent of those who will remain uninsured, according to Urban Institute, a nonpartisan think tank.
And many legal U.S. residents will go without insurance, too. About 36 percent of the population that remains uninsured will qualify for Medicaid but won't sign up for various reasons. Others likely will make too much money to qualify for assistance but be unable to afford coverage.
Here's a look at some of the groups that will likely remain uninsured if the law survives:
ILLEGAL IMMIGRANTS
More than 11 million unauthorized immigrants live in the United States, according to the Pew Hispanic Center, a nonpartisan research center. That amounts to nearly 4 percent of the total population. But there are no provisions that address illegal immigrants in the health care law.
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Health care bill: Millions still go without insurance if law passes high court scrutiny
One of the biggest misconceptions about President Obama's health care overhaul isn't who the law will cover, but rather who it won't.
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A Health Care Mandate That Might Not Matter - Economic Scene
CARLSBAD, Calif., June 19, 2012 /PRNewswire/ -- Life Technologies Corporation (LIFE) today announced it has partnered with The Hospital for Sick Children (SickKids) to advance pediatric genomic research on the Ion Proton Sequencer. Under the agreement, the semiconductor-based platform will be the primary instrument on which multiple clinical research samples will be mapped daily on four sequencers in the hospital's newly launched Centre for Genetic Medicine.
SickKids and Life Technologies will collaborate on developing sequencing workflows and protocols for the Ion Proton System that are tailored for studies of interest to researchers in the Centre. The first collaborative project will focus on sequencing clinical research samples to better understand the genetics behind autism, with a long-term goal to sequence up to 10,000 genomes per year to study various diseases in children.
"The perfect storm of unparalleled advances in genome sequencing technology and information science, and a captivated hospital striving for new ways to move forward in medical treatment, bring us to this important day," says the new Centre's Co-Director, Dr. Stephen Scherer, who also leads The Centre for Applied Genomics at SickKids and the University of Toronto's McLaughlin Centre. "We are very excited to work with Life Technologies to enhance our sequencing capabilities, such that 'genomic surveillance' may soon become the first line of investigation in all clinical research studies ongoing at our institution."
"Since the first published draft sequence of the human genome, our knowledge in genetics has exponentially increased," says Dr. Ronald Cohn, Co-Director of the SickKids Centre for Genetic Medicine. "With the help of this new technology, we will be able to further deepen our understanding of the genetic basis of human disease and translate this directly into daily clinical practice. We have finally reached a point, where individualized medicine is not just a theoretical concept, but will become an integral part of clinical care and management."
The Ion Proton Sequencer is designed to sequence an entire human genome in a day for $1,000. Unlike traditional next generation systems, it relies on semiconductor chips to map human exomes and genomes, making it much faster and less expensive to analyze DNA at unprecedented throughput levels and generate accurate sequencing data.
The Ion Proton Systemis based on the same proven technology as its predecessor, the Ion Personal Genome Machine (PGM), which is designed for sequencing small genomes or sets of genes. Combined with Life Technologies' AmpliSeq targeted sequencing technology, researchers can sequence panels of genes associated with disease on the PGM or exomes and genomes on the Ion Proton Sequencer in just a few hours.
"SickKids has a rich history of being at the forefront of pediatric medicine and we are pleased that its leaders have chosen the Ion Proton Sequencer as the Centre's primary technology to push the boundaries of genomics," said Mark Stevenson, President and Chief Operating Officer of Life Technologies. "Ion semiconductor technology's speed, simplicity and scalability are democratizing sequencing, and it will now be applied in disease research to benefit children."
The above mentioned technology is for research use only and not intended for human diagnostic or therapeutic use.
About Life Technologies Life Technologies Corporation (LIFE) is a global biotechnology company with customers in more than 160 countries using its innovative solutions to solve some of today's most difficult scientific challenges. Quality and innovation are accessible to every lab with its reliable and easy-to-use solutions spanning the biological spectrum with more than 50,000 products for translational research, molecular medicine and diagnostics, stem cell-based therapies, forensics, food safety and animal health. Its systems, reagents and consumables represent some of the most cited brands in scientific research including: Ion Torrent, Applied Biosystems, Invitrogen, GIBCO, Ambion, Molecular Probes, Novex, and TaqMan. Life Technologies employs approximately 10,400 people and upholds its ongoing commitment to innovation with more than 4,000 patents and exclusive licenses. LIFE had sales of $3.7 billion in 2011. Visit us at our website: http://www.lifetechnologies.com.
Life Technologies' Safe Harbor StatementThis press release includes forward-looking statements about our anticipated results that involve risks and uncertainties. Some of the information contained in this press release, including, but not limited to, statements as to industry trends and Life Technologies' plans, objectives, expectations and strategy for its business, contains forward-looking statements that are subject to risks and uncertainties that could cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Any statements that are not statements of historical fact are forward-looking statements. When used, the words "believe," "plan," "intend," "anticipate," "target," "estimate," "expect" and the like, and/or future tense or conditional constructions ("will," "may," "could," "should," etc.), or similar expressions, identify certain of these forward-looking statements. Important factors which could cause actual results to differ materially from those in the forward-looking statements are detailed in filings made byLife Technologies with the Securities and Exchange Commission.Life Technologies undertakes no obligation to update or revise any such forward-looking statements to reflect subsequent events or circumstances.
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Psychiatrists often try two or more medications in a patient suffering with major depression before settling on the one that seems to work best for that individual. Sometimes, after several are tried and abandoned, two (or even three) are used in combination.
Medication selection is part of the art of psychiatry, but, now, testing is available that promises to make it more of a science. A company called AssureRx Health now offers what it calls GeneSightRxpharmacogenomic laboratory testing that helps identify which antidepressants are a good match for a persons genetic makeup, and which are not so good a match.
Sometimes, the testing reveals why three or four antidepressants havent worked for a patient, while pointing in the direction of one that might.
This is extremely good news, because psychiatrists have several different kinds of antidepressants to choose fromsome which increase the activity of the brain chemical messenger serotonin, some which increase the activity of the brain chemical messenger norepinephrine and some which increase both. And they do so by varying mechanisms, requiring the activity of different enzymes.
The technology behind GeneSightRx actually determines which genetic variantsin terms of the enzymes that are activated by antidepressantsa person possesses.
Different antidepressants affect the enzymes very differently. Hence, the testing can literally predict with some accuracy which antidepressants are likely to work in a particular person, and which are likely to cause the fewest side effects.
Recent studies have revealed that antidepressants dont work much better than placebo medications (sugar pills) for many patients. But those studies werent conducted by first selecting patients who are more likely (as determined by GeneSightRx) to respond to the particular medicine being studied.
Its very possible that patients given medications suggested by such testing would do far better than those given placebosbecause they arent being lumped together and given one medicine, regardless of their individual genetic makeup.
Moreover, since many patients discontinue their antidepressants due to side effects like sexual dysfunction and sleeplessness, choosing a medication that is metabolically and genetically less likely to cause these and other side effects makes good sense.
GeneSightRx also predicts which ADHD medications, antipsychotics and pain medications patients are likely to respond to and from which they are likely to experience fewer side effects.
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By Cindy Mindell ~
WEST HARTFORD Its not often that philanthropic donors to a medical cause can see their money pushing researchers over the finish line. But those helping to fund efforts to cure Glycogen Storage Disease (GSD) Type 1a may soon become the rare exception. Last month, A Cure for Sure raised $470,000 toward the $2 million needed to get FDA approval on new gene therapy that has already cured GSD-stricken dogs. The fundraiser was organized and chaired by Gayle and Steve Temkin, and Regan and Barry Stein, two West Hartford couples whose children have the disease. GSD is an inherited disorder caused by the buildup of a complex sugar, glycogen, in the bodys cells. The accumulation of glycogen in certain organs and tissues, especially the liver, kidneys, and small intestines, impairs their ability to function normally. The disease was almost universally fatal until 1971, when the first effective therapy edible cornstarch was discovered. When treated properly, patients are healthy, but inadequate treatment may result in permanent neurologic damage, seizures, or even death.
Dr. David Weinstein
Dr. David Weinstein treats 400 of the estimated 1,100 people afflicted with this type of GSD throughout the world, mostly Jewish. In 2005, he left Harvard Medical School and Boston Childrens Hospital to create the University of Florida Glycogen Storage Disease Program in Gainsville, where he could perform gene therapy on dogs born with GSD. Since then, Weinstein and his research team have cured 10 dogs of the disease at what is the worlds largest clinical and research program for glycogen storage diseases. When the Temkins first brought their daughter, Alyssa, to Weinstein six years ago for lifesaving treatment, she was 15 months old and the doctor had cured one dog. The Temkins went on to establish the Alyssas Angel Fund, which funds travel and treatment for other families with children who have GSD. Every Labor Day weekend for the last three years, the Mandell JCC of Greater Hartford Family Room Parenting Center has sponsored the Big Wheel Derby fundraiser to benefit the fund. Last year, before the event, Weinstein came to West Hartford and met with the Temkins and Steins. We would do anything in our power to support David, says Gayle Temkin. He told us that he needed $2 million to take the gene therapy to the FDA and start human trials. Why couldnt we at least start the fundraising for him? I felt that if I didnt work hard to find a cure and make the effort public, nobody would ever know about it. The Steins approached Alan and Marcia Lazowski, whose son is best friends with their son. According to Temkin, Alan told them, Ive been waiting for an opportunity to help you, and agreed to host a fundraising event. In the wake of A Cure for Sure, the $250,000 goal was nearly doubled by donations from Greater Hartford and Chicago, where the Temkins and Steins hope to organize another fundraiser. People are really feeling that this is the first time ever that theyre part of a cure, Gayle says. For me as a parent, its amazing to be able to say that. Human trials of Weinsteins gene therapy are slated to begin in 2013. For the Temkins, that goal is critical, as Alyssa is the only identified person with GSD who cannot tolerate the cornstarch therapy. But as plans for the fourth annual Big Wheel Derby start to take shape, the fundraiser is infused with new hope and energy, Gayle says. Whats so special now about Alyssas Angel Fund is that, once the cure happens, we can help families without insurance to get the gene therapy. Most of the dogs in Weinsteins program receive two intravenous treatments, at $15,000 each; human subjects will undergo both IV and surgical procedures. So right now, pushing for Alyssas Angel Fund is as important as pushing for A Cure for Sure, she says.
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Close to a cure: Greater Hartford takes on rare Jewish genetic disease
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patients own CD34+ hematopoietic stem cells transduced with bluebird bios lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.
Receiving orphan drug designation is a positive step forward in our efforts to bring hope to ALD patients and their families, said David Davidson, M.D., chief medical officer of bluebird bio. We believe our lentiviral technology has the potential to be a one-time transformative therapy for patients suffering from rare genetic disorders like ALD for whom there are limited treatment options. bluebird is committed to advancing the clinical and commercial development of our gene therapy platform because of the dramatic benefit it may have on the lives of patients.
Orphan drug designation, which is intended to facilitate drug development for rare diseases, provides substantial benefits to the sponsor, including the potential for funding for certain clinical studies, study-design assistance, and several years of market exclusivity for the product upon regulatory approval.
About ALD
Adrenoleukodystrophy (ALD) is a rare X-linked, inherited neurological disorder that, in its most severe form, causes damage to the myelin sheath (an insulating layer of membranes that surrounds nerve cells in the brain) and progressive dysfunction of the adrenal glands. Also known as Lorenzo's Oil disease, ALD is estimated to affect one in every 21,000 boys worldwide. In the childhood cerebral form (CCALD), symptoms usually occur between the ages of 4 and 10. Boys afflicted with this form of ALD develop normally until the onset of symptoms. The symptoms of this disorder often progress rapidly and, in a matter of years, can lead to a vegetative state and, ultimately, death. Current treatment options are limited to allogeneic stem cell transplantation when there is an appropriate donor. Allogeneic transplants carry a significant risk of serious morbidity and death.
About bluebird bio's CCALD Product Development
bluebird bios CCALD product program has the potential to halt the progression of CCALD by providing a functional ABCD1 gene to the patients own stem cells. These stem cells proliferate, and some of the progeny cells travel to the brain where they become microglial cells incorporating the corrective gene. Data from the first clinical study treating X-linked CCALD patients with the companys lentiviral gene therapy product demonstrated continued stable expression of the transgene and the corresponding ABCD-1 protein for over four years in two CCALD patients, resulting in prolonged disease stabilization. bluebird bio plans to initiate a Phase 2/3 clinical study in CCALD in both the United States and Europe in 2013.
About bluebird bio
bluebird bio is developing innovative gene therapies for severe genetic disorders. At the heart of bluebird bios product creation efforts is its broadly applicable gene therapy platform for the development of novel treatments for diseases with few or no clinical options. The companys novel approach uses stem cells harvested from the patients own bone marrow into which a healthy version of the disease causing gene is inserted. bluebird bios approach represents a true paradigm shift in the treatment of severe genetic diseases by eliminating the potential complications associated with donor cell transplantation and presenting a one-time potentially transformative therapy using a patients own stem cells. bluebird bio has two later stage clinical products in development for childhood cerebral adrenoleukodystrophy (CCALD) and beta-thalassemia/sickle cell anemia. Led by a world-class team, bluebird bio is privately held and backed by top-tier life sciences investors, including Third Rock Ventures, TVM Capital, ARCH Venture Partners, Forbion Capital Partners, Easton Capital and Genzyme Ventures. Its operations are located in Cambridge, Mass. and Paris, France. For more information, please visit http://www.bluebirdbio.com.
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