welcome to DuchenneXchange
- a positively charged Duchenne muscular dystrophy community.- join today!
- login
Sarepta Announces Clinical Hold Lifted for its Duchenne Muscular Dystrophy Micro-dystrophin Gene Therapy Program
Sarepta Therapeutics, Inc., a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicine to treat rare neuromuscular diseases, announced today that the Food and Drug Administration (FDA) has lifted the clinical hold for the Company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. Sarepta previously announced on July 25, 2018, that the FDA placed the program on clinical hold due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot. In response, and in collaboration with Nationwide Children’s Hospital, an action plan was developed and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.
rareRelated
-
Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial DesignWave Life Sciences Ltd., a clinical-stag...
-
Exon Skipping Explained from Sarepta Therapeuticshttps://player.vimeo.com/video/238953537...
-
Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patien...This study is done to evaluate whether t...
-
Studying Skeletal Muscle, Heart, and Diaphragm Imaging in Boys With Duchenne Muscular DystrophyBackground: - Duchenne muscular dystrop...
-
New Analyses of Translarna™ (ataluren) Data from ACT DMD Presented at the 12th Annual European Pediatric Neurology...PTC Therapeutics, Inc. today announced t...
-
Safety and Tolerability of Ataluren in a Phase 3 Study of Patients with Nonsense Mutation Duchenne Muscular Dystroph...Objective: Examine the safety/tolerabil...
-
FibroGen Receives Fast Track Designation From the U.S. FDA for Pamrevlumab for the Treatment of Idiopathic Pulmonary...FibroGen, Inc., a biopharmaceutical comp...