President Donald Trump might not care for the World Health Organization’s advice, but that isn’t stopping one of its top ...

RegenXBio has shared updated phase 1/2 data on its Duchenne muscular dystrophy (DMD) gene therapy, providing evidence that ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

An experimental gene therapy for Duchenne Muscular Dystrophy (DMD) has showed better-than-expected results in a three-patient trial, according to preliminary data. Company shares jumped 60 percent ...

INDIANAPOLIS (WISH) — It’s a devastating diagnosis that changes a child’s life forever, Duchenne muscular dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys.

The death of a young boy with Duchenne muscular dystrophy (DMD) who was treated with Pfizer’s experimental gene therapy in a phase 2 trial has prompted the company to pause dosing in another study.

The infant, who was diagnosed with the ... May 14, 2025 — Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene ... Gene Circuits ...

Another fatal case involved a nonambulatory 16-year-old patient with DMD who died 6 days after receiving gene therapy. 20 The patient received fordadistrogene movaparvovec at a dose of 2×10 14 vg ...

PARIS, May 19, 2025--(BUSINESS WIRE)--Genethon unveiled the 2-year follow-up data from its GNT0004 gene therapy clinical trial for Duchenne Muscular Dystrophy (GNT-016-MDYF) at the annual meeting ...

The promise of genome editing to help understand human diseases and create new therapies is vast, but technological ...