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STAT June 21, 2024
Sarepta Therapeutics always wins. That was a given, even before the Food and Drug Administration expanded approval of the company’s Duchenne muscular dystrophy drug on Thursday. Will boys with Duchenne also benefit? I hope they do, but it’s uncertain. And is this a good look for the FDA? No, it is not.
There is much to unpack from the unilateral decision by Peter Marks, the FDA’s top regulator of gene...
