Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

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

in patients with the Duchenne type of dystrophy or in their mothers after a brisk quarter-mile walk. One person (IV-11) examined was a large, muscular man whose serum was obtained on his return ...

A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...

Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy ... helped slow a disease that can rob patients of the ability to walk by ...

Sarepta Therapeutics has decided not to continue the development of vesleteplirsen, a follow-up to its Duchenne muscular dystrophy (DMD) therapy Exondys 51. Digital Sponsored ...

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 ...

UK patients can gain access to Santhera’s as-yet unapproved Duchenne muscular dystrophy drug via an early access scheme. The Medicines and Healthcare products Regulatory Agency granted Raxone ...

A clinical trial investigating oral zinc supplementation in boys with Duchenne muscular dystrophy (DMD ... current nutritional recommendations for patients with DMD. The researchers found that ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.