Duchenne, one of the most severe forms of muscular dystrophy, affects about one out of every 3,600 boys. The inherited rare ...

Despite the push for inclusive education, schools continue to deny admission to differently-abled children, leaving parents ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

A Leicestershire man living with a rare muscle condition has revealed how gaming provides him with the "freedom" to live a ...

Based on the positive findings from the Phase 1/2 study, Genethon is planning to launch Phase 3 testing of GNT0004 next year.

Phase 2 results with Wave Life Sciences' Duchenne muscular dystrophy candidate WVE-N531 ... The improvement in dystrophin was approximately in line with the 5.9% unadjusted improvement seen ...

Ahead of International Disability Day on December 3, many parents complained that their children are not receiving proper ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Despite hotly debated biomarkers and failed or delayed confirmatory trials, the accelerated approval program has a track ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

The company expects the trial to support the submission of a biologics license application with the US Food and Drug Administration in 2026.