Sarep­ta read­ies FDA pitch af­ter a small study spot­lights 1% dy­s­trophin add for golodirsen in Duchenne MD

Af­ter win­ning an FDA ap­proval for Ex­ondys51 as a new ther­a­py for Duchenne mus­cu­lar dy­s­tro­phy with­out hav­ing to ac­tu­al­ly pro­duce da­ta to prove it worked …

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