News

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…

Starting April 1, the public can begin showing their support for Muscular Dystrophy Canada by giving a donation at any of the Canada-based LCBO stores; there are more than 650 throughout Ontario alone. Until April 25, 2015, donation boxes will be displayed at checkout counters. The funds raised from the LCBO Provincial Donation…

The Telegraph‘s Science Editor Sarah Knapton reported last week that the first baby has been born following application of a new In Vitro Fertilization (IVF) technique developed by a team of fertility doctors in London. The new IVF procedure checks embryos for genetic disorders such as the one young…

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…

Sarepta Therapeutics has appointed Edward Kaye, MD, as its new interim Chief Executive Officer (CEO) to lead the  effort to develop their lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD). Serving as CMO since June, 2011, Kaye was in charge of the medical and…

Catabasis, a Cambridge biotech startup, is currently advancing a platform to simultaneously modulate multiple targets in disease pathways. Researchers there are especially focused on diseases like Duchenne muscular dystrophy (DMD) and Amyotrophic lateral sclerosis (ALS). The startup is now in the middle of a fundraise, striving to reach $20.4 million, according…

In a clinical trial titled “…