The company anticipates submitting a biologics licence application for accelerated approval in the US in early 2026.

DMD is caused by mutations in the DMD gene that result in the lack of dystrophin, a protein that protects muscle cells from damage during movements. That lack of dystrophin results in progressive ...

DMD is a devastating genetic condition that primarily affects boys. It causes relentless muscle breakdown, loss of mobility in early adolescence, and premature death. With no cure currently available, ...

Get Instant Summarized Text (Gist) Gene therapy for Duchenne muscular dystrophy (DMD) faces challenges as the immune system may hinder its effectiveness. A large-scale trial showed that the ...

Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy , 2025; DOI: 10.1038/s41434-025-00531-y Cite This Page : ...

raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a large-scale clinical trial.

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, ...

It is the world’s first single-dose agent for gene replacement therapy in Duchenne muscular dystrophy (DMD) patients. The drug is not registered in Russia. «As a result of the negotiations, the price ...

For patients who are ambulatory and have a confirmed mutation in the DMD gene For patients who are non-ambulatory and have a confirmed mutation in the DMD gene. The DMD indication in non-ambulatory ...

Image credit: Shutterstock / Andrii Yalanskyi. Sarepta Therapeutics and Roche are looking to get approval to continue clinical trials of their Duchenne muscular dystrophy gene therapy Elevidys ...

He had claimed to run a lab in Beijing focused on gene-editing treatments for Duchenne muscular dystrophy and Alzheimer’s - motivated, he said, by his mother’s illness. He refused to share ...

Monster Hunter Wilds' Apple of Her Eye quest sends you out to find Alma's glasses, but leaves much of the guesswork up to you to figure out. The first quest marker points you in the right ...