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 ...
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 ...
RGX-202 shows the highest reported microdystrophin levels in ambulatory patients age 8+ among gene therapies. Regenxbio plans to submit a BLA for RGX-202 in mid-2026, with Phase 3 enrollment ongoing.
Copyright 2025 The Associated Press. All Rights Reserved. A sign for the U.S. Food and Drug Administration is displayed outside their offices in Silver Spring, Md ...
A patient has died following treatment with Elevidys, the Sarepta Therapeutics product that is the only FDA-approved gene therapy for Duchenne muscular dystrophy, the company disclosed Tuesday.
One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered ...
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