News

The U.S. Food and Drug Administration has lifted the clinical hold on a Phase 1/2 trial evaluating Sarepta Therapeutics‘ micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy (DMD), the company announced in a press release. On July 25, the FDA placed the…

On Saturday, coaches with the American Football Coaches Association (AFCA) across the U.S. will display patches to promote the annual Coach To Cure MD initiative during college football games. The fundraising effort, in its 11th consecutive year, has raised almost $2 million to fight Duchenne muscular dystrophy (DMD) and has…

More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products Breakthrough Summit. The Oct. 15-16 event, sponsored by the National Organization for Rare Diseases (NORD), takes place at the…

Two new research projects have been funded by the Coalition to Cure Calpain 3 (C3), a nonprofit patient advocacy organization, to develop gene therapies for limb-girdle muscular dystrophy type 2A (LGMD2A). LGMD2A, also known as calpainopathy, is the most common subtype of limb girdle muscular dystrophy (LGMD),…

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial…

Obsessive compulsive disorder (OCD), a type of “internalizing disorder,” are evident in children with Duchenne muscular dystrophy, and particularly associated with anxiety and places considerable stress on the patient’s family, a small retrospective study reported. Researchers call attention to the importance of care…

Using the gene editing process CRISPR/Cas9 enabled a marked restoration of dystrophin production in various muscles of dogs with Duchenne muscular dystrophy (DMD). The study with that finding, “Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy,” was published in the journal…

PTC Therapeutics announced the winners of the 2018 global Strategies to Realize Innovation, Vision and Empowerment (STRIVE) awards, which recognize the efforts of nonprofit organizations working to serve the Duchenne muscular dystrophy community. This year’s winners are patient organizations located in Canada, Argentina, Brazil, Croatia, Hungary, Ireland, Slovenia and Turkey.

Nonprofit Sing Me a Story and Numotion are collaborating to help children with various neuromuscular diseases served by the Muscular Dystrophy Association (MDA) share their unique stories through song. This alliance was supported by the ongoing partnership between MDA and Numotion, a company that provides mobility products and services.

Pfizer is halting the clinical development of domagrozumab (PF-06252616) as treatment for Duchenne muscular dystrophy (DMD) in an ongoing Phase 2 trial. The latest trial data failed to demonstrate domagrozumab’s potential to prevent disease progression or improve patient’s physical capacity, as assessed by mean changes (in seconds) from…