News

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…

BioBlast Pharma Announces Cabaletta Received Fast Track Designation By FDA For Oculopharyngeal Muscular Dystrophy

BioBlast Pharma Ltd., a clinical-stage biotechnology firm focused on orphan disease treatment solutions, recently announced that its intravenous (IV) solution Cabaletta received Fast Track designation from the United States Food and Drug Administration (FDA) to address Oculopharyngeal Muscular Dystrophy (OPMD) patients. Cabaletta is being advanced to address treatment to OPMD, which is an uncommon…

Coalition Duchenne Launches Youtube Interview Series

Newport Beach-based charity Coalition Duchenne recently launched an interview series titled “Making a Difference in Duchenne” through its Youtube channel featuring those who are making a substantial difference in Duchenne muscular dystrophy care, research, education and awareness. Dr. Eduardo Marbán, who is the director of the Cedars-Sinai Heart Institute in Los Angeles, is the…