Marisa Wexler, MS, senior science writer —

Treatment with PF-06939926, an experimental gene therapy for Duchenne muscular dystrophy (DMD), was generally well-tolerated among ambulatory patients — boys able to walk — in a Phase 1B clinical trial. Data also hint at the efficacy of the gene therapy, being developed by Pfizer, with researchers noting that…

The investigational gene therapy SRP-9003 was well tolerated overall and improved measures of physical functioning for up to three years among children with limb-girdle muscular dystrophy type 2E (LGMD2E) in a small clinical trial. “The observed durable treatment effect provides proof-of-concept, and supports our further clinical assessment of…

The corticosteroid Emflaza outperforms prednisone at slowing disease progression in people with Duchenne muscular dystrophy (DMD), according to new research presented at the Muscular Dystrophy Association (MDA) 2022 Annual Meeting being held this week in Nashville, Tennessee. The research was funded by PTC Therapeutics, which markets Emflaza. Corticosteroids…

Treatment with BBP-418 improved walking ability among people with limb-girdle muscular dystrophy type 2i (LGMD2i) in a Phase 2 clinical trial, early data show. “To date, people with LGMD2i have no approved disease-modifying treatment options. Many of these patients see their quality of life deteriorate rapidly and lose their…

A potentially pivotal Phase 3 clinical trial called HOPE-3, which will test the effectiveness of the experimental cell therapy CAP-1002 as a treatment for Duchenne muscular dystrophy (DMD), is expected to launch soon. Capricor Therapeutics, the company developing CAP-1002, announced that the U.S. Food and Drug Administration…

A protein called GCN5 is vital for normal muscle health and helps to coordinate the expression of several important muscle proteins, including dystrophin, according to a study done in mice. The results suggest that targeting GCN5 may be a useful therapeutic avenue in diseases of the muscles, such as…

Children with Duchenne muscular dystrophy (DMD) who had been given a placebo in a clinical trial experienced a marked improvement in motor abilities after being treated with Sarepta Therapeutics‘ experimental gene therapy SRP-9001, new top-line data show. “We are delighted to report positive results for Part 2 of…

Elamipretide, an investigational therapy designed to increase the activity of mitochondria, may boost the effectiveness of exon-skipping therapies for Duchenne muscular dystrophy (DMD), its developer, Stealth BioTherapeutics, reported. “Our new preclinical data increases our optimism that elamipretide may help address the unmet need for therapies in DMD,” Reenie…

Atamyo Therapeutics has been given the go-ahead to start clinical testing of ATA-100, the company’s investigational gene therapy for a specific form of limb-girdle muscular dystrophy (LGMD) called type R9 (LGMDR9). With approval from the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA), Atamyo now plans to start dosing…

Santhera Pharmaceuticals and ReveraGen BioPharma are gearing up to submit a new drug application (NDA) asking for U.S. approval of vamorolone as a treatment for Duchenne muscular dystrophy (DMD). The companies announced Tuesday they had completed a pre-NDA meeting with the U.S. Food and Drug Administration (FDA).