News

BioMarin Concludes New Drug Application Proposal for Drisapersen for Duchenne Muscular Dystrophy

BioMarin Pharmaceutical Inc., a company that develops and commercializes biopharmaceuticals for serious diseases and medical conditions, recently announced conclusion of the continuing proposal of a New Drug Application (NDA) to the FDA for drisapersen, an investigational exon-skipping drug candidate aimed at treating the largest genetically defined subset of…

PhaseBio Announces Positive Data For Duchenne Muscular Dystrophy therapy in Pre-Clinical Mice Models

PhaseBio Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company that develops novel drugs to treat diabetes and cardiovascular dysfunction in patients suffering from rare diseases, recently announced positive data from a pre-clinical study for the use of PB-1046 in Duchenne Muscular Dystrophy (DMD) mice models. The results were presented in…

Toronto Walk To Support Muscular Dystrophy

Toronto will come together on Saturday, May 2nd, to make muscles move against muscular dystrophy. Thousands of people suffering with any kind of neuromuscular disorder in Canada will be supported by the community at the annual Toronto Walk for Muscular Dystrophy. Mayyan Ziv, who is the 2015 Toronto Walk Ambassador, strongly believes in the…

Milo Treats First DMD Patients with Novel Follistatin Gene Therapy

Clinical stage startup company Milo Biotechnology has begun treating the first Duchenne muscular dystrophy (DMD) patients with follistatin gene therapy. The treatment is being provided through intramuscular injections and its effects in maintaining or restoring muscle function are being studied at Nationwide Children’s Hospital. The research team, which…

Santhera Receives FDA Fast Track Designation For Raxone®/Catena® To Treat Duchenne Muscular Dystrophy

Santhera Pharmaceuticals recently announced it received Fast Track designation from the United Stated Food and Drug Administration (FDA)  for its Raxone®/Catena® (idebenone) to address treatment for Duchenne Muscular Dystrophy (DMD). Through the FDA’s Fast Track program, both the development and review of the drug are facilitated, since the has the potential to treat a severe condition, fill unmet medical…