Critics have excoriated Sarepta Therapeutics (SRPT) for winning FDA approval for a drug to treat Duchenne muscular dystrophy despite scant scientific proof that it helps patients suffering from the inherited, muscle-wasting disease.
Sarepta has long insisted the doubters are wrong. And on Wednesday, the biotech firm strengthened its case with data from a new clinical trial: A second Sarepta drug, designed to treat another form of Duchenne muscular dystrophy, is producing significantly larger quantities of dystrophin, a crucial muscle protein normally missing in these patients.
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