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Research
July 24, 2018
Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
Research
April 2, 2018
Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.
News
September 20, 2016
Sarepta Therapeutics announced yesterday that the FDA has granted accelerated approval for eteplirsen for the treatment of Duchenne muscular dystrophy...