The U.S. Food and Drug Administration (FDA) and the European Commission (EC) have granted Orphan Drug designations to Solid Biosciences’ SGT-001, a micro-dystrophin gene therapy under development for the treatment of Duchenne muscular dystrophy (DMD). Clinical trials should commence in 2017, the company announced…
The Muscular Dystrophy Association (MDA) and RYR-1 Foundation have announced an agreement to advance research and clinical care, raise awareness and improve patient, medical and public education about RYR1-related myopathies. The partnership aims to enhance the quality, quantity and scope of basic, translational and clinical research, bolster…
The Muscular Dystrophy Association (MDA) will partner with the creative agency 50,000feet for the fifth year to help the nonprofit raise money for new research and therapies for neuromuscular diseases. The MDA will host a 2016 Muscle Team fundraiser Oct. 21 in Chicago, and 50,000feet’s brand expertise will be crucial to advance the…
The nonprofit Parent Project Muscular Dystrophy (PPMD) has awarded $239,000 to Dr. Terence Partridge, with the Research Center for Genetic Medicine at Children’s National Medical Center. The grant will support ongoing work aimed at a better understanding of exon skipping therapies for Duchenne muscular dystrophy (DMD), and their validation in future clinical trials. DMD is caused by mutations in…
Catabasis Pharmaceuticals recently completed its target enrollment of 30 patients for the open-label extension of the Phase 2 portion (Part B) of the MoveDMD clinical trial evaluating edasalonexent (CAT-1004) in boys with Duchenne muscular dystrophy (DMD). Edasalonexent is an oral drug that researchers hope will revolutionize DMD treatment. Edasalonexent…
Elixirgen has announced the launch of its new Quick-Tissue 1.0 Series for use by scientists exploring new ways of treating muscle-degenerative diseases like muscular dystrophy (MD). The biopharmaceutical company, located at Johns Hopkins campus, specializes in stem cell biology. The Quick-Tissue 1.0 Series includes a skeletal muscle differentiation kit called…
Idera Pharmaceuticals recently presented preclinical data regarding the gene-silencing mechanisms of its third generation antisense (3GA) technology platform, a potential therapeutic for diseases that include, but are not limited to, facioscapulohumeral muscular dystrophy (FSHD). The presentations took place at the recent 12th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal,…
Capricor Therapeutics, Inc. announced that the U.S. Department of Defense (DoD) has awarded the company a $2.4 million grant to establish a scalable, commercially-ready process to manufacture CAP-2003, the company’s proprietary exosome product candidate. Capricor is a clinical-stage biotechnology company working on the discovery, development and commercialization of biological therapies.
The U.S. Food and Drug Administration (FDA) has allowed an investigational new drug (IND) application for Carmeseal-MD in Duchenne muscular dystrophy (DMD), according to Phrixus Pharmaceuticals. Carmeseal-MD (Poloxamer-188 NF, or P-188 NF) is the first disease-modifying agent in diseases characterized by membrane instability such as Duchenne MD or limb…
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Mallinckrodt’s investigational new drug (IND) application for Synacthen Depot to treat Duchenne muscular dystrophy (DMD). The invesigational drug is a depot formulation (a sustained-action drug formulation that allows slow release and gradual absorption) of Synacthen (tetracosactide), a synthetic…
Get regular updates to your inbox.