Duchenne muscular dystrophy is a debilitating genetic disorder that has garnered much attention lately as the U.S. Food and Drug Administration prepares to review two experimental drugs that could soon treat some patients with the degenerative muscle disease.
A team of researchers at the University of Michigan Health System identified a novel method of triggering the “instructions” normally given to cells by the muscle protein dystrophin, which is found in cardiac muscles cells and in muscles used for movement. This new method could produce a key to a breakthrough therapeutic strategy for patients with Duchenne Bekcer muscular dystrophy.
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical stage drug development company built on a pathway pharmacology technology platform, today announced that the European Commission (EC) has granted
orphan medicinal product designation to CAT-1004 for the
treatment of Duchenne muscular dystrophy (DMD).
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) will review the New Drug Application (NDA) for drisapersen. The FDA is currently reviewing drisapersen for the treatment of patients with Duchenne muscular dystrophy amenable to exon 51 skipping. The advisory committee will review drisapersen data included in a new drug application (NDA) during a meeting on November 24, 2015.
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced results from the Phase 3, double-blind, placebo-controlled, 48-week ACT DMD trial of Translarnaâ„¢ (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The trial results showed clinically meaningful benefits for Translarna-treated patients.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) is tentatively scheduled to review Sarepta’s New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, on January 22, 2016.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced additional clinical efficacy and safety data from the Company’s Phase IIb program of eteplirsen in patients with Duchenne muscular dystrophy (DMD).
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has filed the New Drug Application (NDA) for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Approximately 13% of people with Duchenne muscular dystrophy are estimated to have a mutation addressable by Eteplirsen/exon 51 skipping.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative RNA-targeted therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for eteplirsen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to skipping exon 51. The Rare Pediatric Disease Designation supplements the Orphan Drug Designation and Fast Track Status previously granted by the FDA for eteplirsen.
