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 ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies 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 ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...

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

REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...

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 ...