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Santhera Pharmaceuticals has asked the European Medicines Agency (EMA) to approve vamorolone to treat Duchenne muscular dystrophy (�鶹��). 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 (�鶹��) 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 (�鶹��), 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 (�鶹��) and raising research funds for a cure. Slated for Sept. 24, the 15th annual event is a…

The Muscular Dystrophy Association (�鶹��) 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 �鶹�� 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 (�鶹��), 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 (�鶹��), maintained biomarkers of bone formation and turnover compared to prednisone, according to the now published details of the VISION-�鶹�� study. Standard anti-inflammatory corticosteroids, such as prednisone, have been shown to stunt growth with long-term use. Vamorolone is a…