Sarepta's DMD candidate leads to microdystrophin expression of 76.2% in Phase I/IIa

Sarepta Therapeutics Inc. (NASDAQ:SRPT) reported on June 19 preliminary data from a Phase I/IIa trial evaluating AAVrh74.MHCK7.micro-Dystrophin to treat Duchenne muscular dystrophy (DMD).

In three DMD patients in the open-label, U.S. trial, the microdystrophin gene therapy led to mean sarcolemma-localized transduced microdystrophin

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