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Podcast: γ¬γ’γΈγ§γθ±δΌθ©± Daily News Article Podcast
Episode: First gene therapy for deadly form of muscular dystrophy gets FDA approval for young kids
Description: The first gene therapy for a deadly form of muscular dystrophy received preliminary U.S. approval despite concerns from some government scientists about the treatment's ability to help boys with the inherited disease.
The Food and Drug Administration approval provides a new option for some patients with Duchenne muscular dystrophy, a rare muscle-wasting disease that causes weakness, loss of mobility and early death. It almost always affects males.
Drugmaker Sarepta Therapeutics said it would charge $3.2 million for the one-time treatment, slightly less than a $3.5 million gene therapy for hemophilia launched last year. Like most medicines in the U.S., the...