Wave Life Sciences Initiates Clinical Trial for Lead Program in Duchenne Muscular Dystrophy (DMD). First clinical trial of WVE-210201, an exon 51 skipping investigational therapy, in ambulatory and non-ambulatory boys 5 to 18 years old.
The Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) has issued a complete response letter stating it cannot approve PTC Therapeutics, Inc.'s New Drug Application (NDA) for the investigational medicine ataluren. Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with Duchenne caused by a nonsense mutation.For more, please see PTC Therapeutics, Inc.'s full press release or President and CEO Stuart Peltz, Ph.D.'s community letter.
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The following sites Roche's Phase 2/3 Study of RG6206 have opened for enrollment.
Dear Duchenne Community
On behalf of all of PTC, we want to thank all of you for supporting the advisory committee review of ataluren.
Dear Duchenne community,
We at Roche and our U.S. affiliate Genentech are privileged to take on development of BMS-986089. Moving forward this investigational molecule will be called RG6206. Our team is looking forward to partnering with the Duchenne community to make a meaningful difference to young people with Duchenne.
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.
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.
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).
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™).
Pfizer Inc. announced today the completion of patient enrollment in a multicenter Phase 2 clinical trial of the investigational compound, domagrozumab (PF-06252616), in boys with Duchenne muscular dystrophy (DMD).
Yesterday, PTC Therapeutics announced their plans to launch EMFLAZA (deflazacort) in the coming weeks for a list price of $35,000 per 25 kilograms.
Bristol-Myers Squibb is testing a new anti-myostatin adnectin protein targeting Duchenne Muscular Dystrophy. BMS-986089 decreases myostatin, which is known to slow muscle growth.