News

Treatment with Viltepso (viltolarsen) helps preserve motor function in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to four-year data from an open-label extension study. “These data represent the longest clinical experience of an exon 53 skipping therapy for the treatment of Duchenne…

EDG-5506, Edgewise Therapeutics’ experimental oral therapy, continues to safely reduce muscle damage and improve physical function in adults with Becker muscular dystrophy (BMD), according to six-month interim data from the Phase 1 ARCH trial. Treatment led to a marked reduction in the average levels of key markers of muscle…

The Muscular Dystrophy Association (MDA) will host the MDA Tribute Awards Oct. 27 in Boston to honor four people who have contributed to progress in muscular dystrophy research and care, and in lobbying efforts on behalf of those with neuromuscular disorders, particularly Duchenne muscular dystrophy (DMD). The awards…

The U.K.’s National Institute for Health and Care Excellence (NICE) has published new draft guidance that does not recommend Translarna (ataluren) be covered to treat Duchenne muscular dystrophy (DMD) caused by nonsense mutations. According to NICE, although there’s evidence Translarna can benefit people with DMD, it’s not worth…

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…