Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

Barry Byrne, MD, PhD, Powell Gene Therapy Center at the University of Florida, discusses gene therapy considerations for pediatric patients and how newborn screening can influence outcomes for ...

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

Dyne Therapeutics Inc. is advancing novel therapeutics for people living with genetically driven neuromuscular diseases.

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

Previous studies have shown that protein expression of DOCK7 is increased in skeletal muscle biopsies from patients with Duchenne muscular dystrophy (DMD), leading researchers from the University of ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

Wave Life Sciences’ Phase II open-label trial investigating its disease-modifying drug for boys living with Duchenne Muscular ...

The firm will seek accelerated approval for WVE-N531 in DMD amenable to exon 53 skipping based on FDA feedback and Phase II data.

After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent Project Muscular Dystrophy stepped up—as they always do. I’ve always ...

an investigational gene therapy for Duchenne muscular dystrophy. Results were presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. At dosing ...

In 2023, Elevidys received expedited U.S. approval despite concerns from some Food and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the first ...