News

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 “…

In a recent study titled “Long Term Natural History Data in Ambulant Boys with Duchenne Muscular Dystrophy: 36-Month Changes,” a research team from the Department of Pediatric Neurology at the Catholic University in Rome, Italy examined the changes at 3 years follow-up in a 6-minute walk test…

The Muscular Dystrophy Association (MDA) recently announced that MDA clinics are offering genetic testing for those with limb-girdle muscular dystrophy (LGMD) thanks to a grant from Sanofi company Genzyme in collaboration with Emory Genetics Laboratory. This represents a major step in diagnostic advances that will help those with the disease and their respective clinicians so they can find…

Patients in Indiana who suffer from terminal diseases such as muscular dystrophy may now have access to experimental drugs that have not yet been approved by the U.S. Food and Drug Administration (FDA), but are thought to be beneficial in reducing the burden of the disease or improving patients’…