|Gene therapy cocktail shows promise in long-term clinical trial for rare fatal brain disorder|
|Wednesday, December 19, 2012|
Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.
A report of the trial appears in the Dec. 19, 2012 online edition of the journal Science Translational Medicine.
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CAROLINA IN THE NEWS
Obama defends Obamacare: 'We will make this work'
President Barack Obama on Tuesday launched a planned three-week-long push for — and defense of — his embattled health-care reform law, vowing to "make this work"… But Dr. Sam Weir, a physician cited by Obama in his speech for his support of the Affordable Care Act, told CNBC.com that "many of my patients are looking forward to getting coverage."…"From where I sit, anything that expands coverage of people who have to make choices about what medicines to take because they can't afford them, or they end up in the emergency room because haven't gotten the preventative care they need, those are important steps in terms of health care policy," said Weir, medical director of family medicine at the University of North Carolina School of Medicine.