Parent Project Muscular Dystrophy (PPMD) has granted $465,000 to Renzhi Han, PhD, a professor at the Ohio State University College of Medicine, to advance research into a targeted gene editing approach that may protect cardiac and skeletal muscle function in people with Duchenne muscular dystrophy (DMD).
Nippon Shinyaku filed a request for the approval of viltolarsen (NS-065/NCNP-01) to treat Duchenne muscular dystrophy amenable to exon 53 skipping with the U.S. Food and Drug Administration (FDA), the company announced in a press release. The request came in the form of a rolling new drug application (NDA) whose…
Sarepta Therapeutics is acquiring an investigational gene therapy program focused on calpain-3 for the treatment of limb-girdle muscular dystrophy type 2A (LGMD2A). The candidate therapy was developed by the Research Institute at Nationwide Children’s Hospital. It is Sarepta’s sixth gene therapy candidate for limb-girdle MD, which causes…
Long-term treatment with Raxone (idebenone) slows down loss of respiratory function in Duchenne muscular dystrophy (DMD) patients for up to six years, according to recent results from Santhera Pharmaceuticals’ SYROS trial. The company is recruiting participants for the SIDEROS-E Phase 3 study (NCT03603288). It aims to assess the long-term safety…
Capricor Therapeutics announced that patient dosing  has resumed in its HOPE-2 trial of CAP-1002, an investigative cell therapy for Duchenne muscular dystrophy The company placed a voluntary hold on dosing after one patient had a serious allergic reaction during blind testing in December, to allow for an…
Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School of Medicine to advance research into a mini-dystrophin gene therapy that may protect cardiac function in Duchenne muscular dystrophy (DMD) patients. The funds were raised during PPMD’s Cardiac Initiative holiday campaign highlighting the…
The University of Virginia (UVA) Children’s Hospital has been named a Certified Duchenne Care Center by Parent Project Muscular Dystrophy (PPMD), making it the 22nd such center recognized by the patient advocacy group for the quality and comprehensiveness of its neuromuscular program. UVA’s hospital is also the first CDCC with a prominent…
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…
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.
Nicholas Johnson, vice chair of research and associate professor of neurology at Virginia Commonwealth University in Richmond, was awarded a $700,000 grant from the Muscular Dystrophy Association (MDA) to establish the Limb-Girdle Muscular Dystrophy (LGMD) Clinical Research Network…
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