Tag Archives: dystrophin gene

September, 2016

19 September
Sarepta’s Eteplirsen Wins FDA Approval for Treatment of Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent …
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9 May
WAVE Life Sciences to Advance Next-Generation Nucleic Acid Therapies to Address Unmet Need in DMD

CAMBRIDGE, Mass.–(BUSINESS WIRE)–WAVE Life Sciences Ltd. (NASDAQ:WVE), a genetic medicines company focused on developing stereopure nucleic acid therapies for patients impacted by rare diseases, today reaffirmed its commitment to advance next-generation nucleic acid therapies to address the significant unmet need of patients diagnosed with Duchenne Muscular Dystrophy (DMD). WAVE’s DMD …
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