News

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…

Santhera Pharmaceuticals announced in a press release that an international team of researchers recently published in the journal The Lancet the results of the DELOS Phase 3 trial validating the efficacy and safety of Santhera’s Raxone/Catena (idebenone) in patients with Duchenne muscular dystrophy (DMD). The study…

Japan’s Cyberdyne, Inc. has applied for marketing authorization of their HAL for Medical Use (Lower Limb Type) robot exoskeleton as a new medical device for orphan neural-muscular diseases, such as muscular dystrophy, amyotrophic lateral sclerosis (ALS) and others under Japan’s Pharmaceutical and Medical Device Law (PMDL). Under the law,…

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…

GlobalData recently reported that the global treatment market for Duchenne Muscular Dystrophy will expand in value at a staggering Compound Annual Growth Rate (CAGR) of 160.5%, from about $8.2 million in 2014 to $990 million by 2019. In the latest report, OpportunityAnalyzer: Duchenne Muscular Dystrophy – Opportunity and Market Analysis…

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 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…

Researchers at Sun Yat-sen University, China recently published in the journal Disease Markers a new method to help assess muscular dystrophy disease severity and progression. The study is entitled “Serum Creatinine Level: A Supplemental Index to Distinguish Duchenne Muscular Dystrophy from Becker Muscular…

According to recent study, progressive myocardial fibrosis is associated with a decline in the left ventricular function in patients with Duchenne muscular dystrophy. Longer steroid treatment duration is also associated with a lower age‐related increase in myocardial fibrosis burden, according to the study now published in the journal…

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…