Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

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 ...

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.

Satellos said that it will be advancing its DMD small molecule to a Phase II trial, following the Phase I data.

Apr. 8, 2025 — A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the ... New Gene Editing Tool Shows ...

Capricor's lead asset, deramiocel, targets DMD-related cardiomyopathy and faces key FDA milestones. Find out why CAPR stock ...

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 ...

Low-dose interleukin-2 as an add-on therapy to riluzole (Rilutek) led to a non-significant reduction in mortality in an ...