Jun 19 2018 An experimental gene therapy appeared to dramatically increase the production of a muscle-making protein in three young boys with Duchenne muscular dystrophy a deadly and irreversible disease according to results being presented by the drugmaker Sarepta Therapeutics at an investor event this morning.Sarepta Therapeutics Announces that at its First R&D Day Jerry Mendell M.D. Presented Positive Preliminary Results from the First Three Children Dosed in the Phase 12a Gene Therapy Micro-dystrophin Trial to Treat Patients with Duchenne Muscular DystrophyDuchenne Muscular Dystrophy (DMD) is the most common neuromuscular disease in child affecting approximately one in every 3500 male births. It is caused by mutations in the gene for dystrophin which corresponds to a protein that has an extremely important role in muscle fiber integrity and function.
Gene therapy for a specific disease is not a one-and-done scenario. Several different types of mutations can affect a given gene or set of genes that regulate a LGMD2D is an autosomal recessive form of muscular dystrophy mainly affecting the proximal muscles and resulting in difficulty walking. Most individuals have onset in childhood the disorder is progressive.MDA Venture Philanthropy is the Muscular Dystrophy Associations drug development program which is exclusively focused on funding the discovery and clinical application of treatments and cures for neuromuscular diseases.
Solid Biosciences will resume their Phase 12 clinical trial for their investigational Duchenne muscular dystrophy (DMD) treatment SGT-001 after the Food and Drug Administration (FDA) removed a hold on the trial. SGT-001 is a novel adeno-associated viral (AAV) vector mediated gene transfer that Sarepta Therapeutics presented preliminary data for its experimental treatment for patients with Duchenne muscular dystrophy. Duchenne muscular dystrophy is a rare genetic disease that causes muscle wasting and can be fatal before patients turn 30.AveXis Inc. and Genethon today announced they have entered into an exclusive worldwide license agreement for in vivo gene therapy delivery of AAV9 vector into the central nervous system (CNS) for the treatment of spinal muscular atrophy (SMA).About AFM-Tlthon. The French Muscular Dystrophy Association (AFM-Tlthon) is composed of patients and their families who are affected by genetic rare and progressive diseases that lead to heavy handicaps.