Among women who carry mutations that can cause Becker muscular dystrophy (BMD) or…
Articles by Marisa Wexler, MS
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen…
No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may…
An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children…
The Phase 3 portion of a clinical trial testing RGX-202, an investigational gene therapy for Duchenne muscular dystrophy (DMD),…
Duchenne Parent Project Spain is investing €247,000 (just over $290,000 USD) in a project that aims to create a…
Epicrispr Biotechnologies is partnering with Forge Biologics to support the development of EPI-321, Epicrispr’s one-time epigenetic treatment candidate…
Regulators in the European Union have granted orphan drug designation to SGT-003, a gene therapy for Duchenne muscular dystrophy…
The Committee for Medicinal Products for Human Use (CHMP) has recommended that approval of Agamree (vamorolone) in the European…
Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of…