Industry Partner News: Sarepta Therapeutics Announces Positive Dystrophin Results in Study Evaluating Golodirsen (SRP-4053)

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53.

Industry News: FDA Announces Advisory Committee Meeting to Review Ataluren.

The Food and Drug Administration (FDA) has published the date, location, and time of the Central and Peripheral Nervous System (PCNS) Advisory Committee Meeting to review autaluren and request for public comments to the Federal Register. The Advisory Committee will discuss  ataluren's new drug application by PTC Therapeutics, for the treatment of patients with Duchenne muscular dystrophy due to a nonsense mutation in the dystrophin gene.

Industry News: Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy

Industry News: Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc.a biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases, today announced that it has received official minutes of the meeting held recently between the U.S. Food and Drug Administration (FDA) and Capricor to discuss the development of intravenous CAP-1002 (allogeneic cardiosphere-derived cells) for the treatment of Duchenne muscular dystrophy (DMD).

Industry News: PTC Announces FDA Advisory Committee Meeting for Ataluren

today announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). today announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™).