Sarepta Therapeutics, based in Cambridge, Massachusetts, focuses on RNA-targeted therapeutics for rare diseases, particularly Duchenne muscular dystrophy, and has developed four approved products. The company employs 1,314 people and has about 40 programs in its pipeline.
SRPT has been in the news recently: The FDA has placed a clinical hold on Sarepta Therapeutics Inc.'s SRPT gene therapy trials for limb girdle muscular dystrophy. Additionally, George F. Tidmarsh has been appointed as the director of the Center for Drug Evaluation and Research (CDER) by the FDA.
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