The U.S. health regulator has granted accelerated approval to Sarepta Therapeutics' first-of-its-kind gene therapy for Duchenne muscular dystrophy (DMD), an inherited progressive muscle-wasting disorder that almost always affects young boys.
first-of-its-kind gene therapy for Duchenne muscular dystrophy , an inherited progressive muscle-wasting disorder that almost always affects young boys.
Elevidys could change the way that DMD patients, who rarely survive beyond their thirties, are treated. Currently, approved steroids and some therapies for specific genetic mutations only control symptoms and slow disease progression. Current options for DMD patients are limited. Of four new "exon-skipping" therapies - intended for a smaller group of patients with specific genetic mutations - three are from Sarepta.
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