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Sarepta is a global biotech company that develops gene therapy, RNA technologies, and gene editing for rare neuromuscular and central nervous system diseases. Learn about their science, pipeline, treatments, and careers.
- Investors
Sarepta Therapeutics Announces Inducement Grants Under...
- Leadership
"I approach my work every day with an open mind, a...
- Limb-girdle Muscular Dystrophy
Sarepta’s gene therapy program in development uses distinct...
- Charcot-Marie-Tooth Disease
This gene therapy program, which targets CMT1A, the major...
- Investors
Sarepta is a global biotechnology company that aims to engineer precision genetic medicine for rare diseases that devastate lives and futures. Learn about their mission, leadership, patient affairs, grants, and locations.
Jun 22, 2023 · ELEVIDYS is the first gene therapy to treat Duchenne muscular dystrophy, a rare genetic disease that causes muscle degeneration. The FDA approved ELEVIDYS based on increased expression of ELEVIDYS micro-dystrophin protein in skeletal muscle, but requires a confirmatory trial.
Douglas S. Ingram. ( CEO & President) Revenue. $301 Million (2018) Website. www .sarepta .com. Sarepta Therapeutics, Inc. ( Nasdaq : SRPT) is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States.
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Find the latest news and updates from Sarepta, a biotech company developing gene therapy for rare neuromuscular diseases. Read press releases, stories and perspectives from advocates, innovators, patients and scientists.
Feb 16, 2024 · Sarepta Therapeutics announced that the FDA has accepted and filed its efficacy supplement to convert the accelerated approval of ELEVIDYS, a gene therapy for Duchenne muscular dystrophy (DMD), to a traditional approval. The supplement aims to remove the age and ambulation restrictions and broaden the indication to include all DMD patients with a confirmed mutation in the DMD gene.