First gene therapy for deadly form of muscular dystrophy gets FDA approval for young children
First gene therapy for deadly form of muscular dystrophy gets FDA approval for young children
www.pbs.org First gene therapy for deadly form of muscular dystrophy gets FDA approval for young children
The treatment from Sarepta Therapeutics was approved Thursday for children ages 4 and 5 with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes early death.
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Interesting discussion in the article. Seems like it's a pretty experimental therapy atm without any conclusive evidence that it works.