News

Biopharmaceutical company PTC Therapeutics was honored with the Outstanding Innovation Award at The 2014 Rare & Orphan Advocacy & Research Awards. The prize was granted to PTC for their lead therapy Translarna, which has recently become the first treatment for Duchenne muscular dystrophy to be approved in the European…

The International Duchenne Alliance has announced that it will provide a $500,000 grant to researchers Jerry Mendell, M.D. and Milo Biotechnology to support their work in a first ever follistatin gene therapy trial for Duchenne muscular dystrophy. The study will be held at Mendell at Nationwide Children’s Hospital in Columbus, Ohio, and the…

A team of researchers recently presented their latest results showing how injecting cardiac stem cells in mouse models of Duchenne muscular dystrophy can reverse damage to the heart. These findings carry a new therapeutic promise to patients with muscular waisting disorders. The study was presented in the American Heart…

The national nonprofit organization CureDuchenne and biotechnology company Prosensa Holding, both of which are committed to finding a cure for Duchenne muscular dystrophy (DMD) in different ways, recently presented a collaborative project at the 6th annual Partnering for Cures event. The conference, which is wrapping up today in New York City, gathered about 1,000…

Researchers throughout the world continue to work towards improved treatments for Duchenne muscular dystrophy (DMD). In response to recent advancements in the lab, the Muscular Dystrophy Association has published their Fall 2014 drug development update, highlighting the most prominent and promising DMD research and drug development. Translarna (ataluren),…

A study entitled “Forelimb Treatment in a Large Cohort of Dystrophic Dogs Supports Delivery of a Recombinant AAV for Exon Skipping in Duchenne Patients” describes the positive results of using a recombinant adeno-associated virus vector to restore dystrophin levels in dogs affected with Duchenne muscular dystrophy.

Biopharmaceutical company Sarepta Therapeutics, which specializes in developing therapies for rare and infectious diseases, has been waiting for approval from the U.S. Food and Drug Administration (FDA) for its new drug treatment for muscular dystrophy. However, the FDA has decided to delay the decision, and has asked the company for more research. The FDA…

A group of investigators from the Massachusetts Institute of Technology (MIT) recently joined forces with the Myotonic Dystrophy Foundation (MDF) and Parent Project Muscular Dystrophy (PPMD) to host the second annual “Strength, Science, & Stories of Inspiration” event, which took place last Friday, November 7 at the MIT Museum in…

Almost $800,000 in funding was recently awarded to Murdoch University researchers from the Centre for Comparative Genomics (CCG) to develop genetic drugs to treat rare diseases. The money comes from the National Health and Medical Research Council (NHMRC) and will allow for the expansion of successful research into Duchenne Muscular Dystrophy…

Investigators from the Helmholtz Zentrum München and Ludwig-Maximilians-Universität München in Germany are organizing a crowd sourcing exercise as a way of determining new algorithms that they expect will be able to accelerate their research into treatment for amyotrophic lateral sclerosis (ALS). The challenge itself, as well as the most effective algorithms found, were recently published…