News

Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (DMD). The agency’s assessments of new approval requests — in the form of marketing authorization applications — take up to 210 days, but can be longer if the Committee for Medicinal Products…

A form of congenital muscular dystrophy caused by mutations in a gene called GGPS1 may not always lead to hearing loss, contrary to what has been described previously, according to a recent study. The report, “GGPS1-associated muscular dystrophy with and without hearing loss,” was published as a brief…

Sarepta Therapeutics has submitted an application to the U.S. Food and Drug Administration (FDA) asking the agency to grant accelerated approval to the gene therapy SRP-9001 (delandistrogene moxeparvovec) as a treatment for Duchenne muscular dystrophy (DMD) in patients who are able to walk. “Every hour of every…

Dystrogen Therapeutics’ experimental cell therapy, DT-DEC01, continues to show signs of efficacy in three boys with Duchenne muscular dystrophy (DMD), who exhibited improved muscle strength and motor function after six months in a clinical trial. All patients showed signs of clinical improvements irrespective of their mutation in the…

A first patient has been dosed in Denmark as part of a Phase 1/2 trial of ATA-100, Atamyo Therapeutics’ one-time gene therapy for a specific form of limb-girdle muscular dystrophy called LGMD-2I or LGMD-R9. “This is an exciting milestone for our company but most importantly, if this clinical trial is…

The beginning of a new college football season also means it’s time for Coach to Cure MD (CTCMD) — a campaign aimed at calling attention to Duchenne muscular dystrophy (DMD) and raising research funds for a cure. Slated for Sept. 24, the 15th annual event is a…

The Muscular Dystrophy Association (MDA) has awarded more than $17 million to projects aimed at advancing research and developing new treatments in muscular dystrophy (MD) and other neuromuscular diseases. The funding adds to the more than $1 billion the MDA has already invested in research. “The Muscular Dystrophy…

Four months of treatment with the experimental oral therapy EDG-5506 reduced markers of muscle damage and improved measures of physical function among men with Becker muscular dystrophy (BMD) in the Phase 1 ARCH clinical trial, new data show. Edgewise Therapeutics, the company developing EDG-5506, recently launched a Phase…

The first patient has received Dyne Therapeutics‘ investigational therapy DYNE-251 for treating Duchenne muscular dystrophy (DMD), the company has announced. The therapy is being tested in the DELIVER Phase 1/2 clinical trial and is indicated for patients with mutations amenable to exon 51 skipping. “We are excited to…

Vamorolone, an investigational therapy for Duchenne muscular dystrophy (DMD), maintained biomarkers of bone formation and turnover compared to prednisone, according to the now published details of the VISION-DMD study. Standard anti-inflammatory corticosteroids, such as prednisone, have been shown to stunt growth with long-term use. Vamorolone is a…