It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the current understanding of the genetic factors behind muscular dystrophy.

This year's Futures National Conference, hosted by CureDuchenne for the Duchenne and Becker MD communities, is scheduled to ...

Indicated for patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, the drug could be used in around 13% of the disease population. Sarepta’s share price almost ...

Families say it's 'completely frustrating' that children with Duchenne Muscular Dystrophy are being denied access to free ...

Explore GISTs, rare GI tumors. Discover key details on genetic causes, diagnostic methods, and cutting-edge treatment ...

Homeowners insurance is the best way to protect your biggest investment, but not everyone gets approved for a policy. You may be rejected for a variety of reasons, from having bad credit to living ...

It offers EXONDYS 51 injection to treat Duchenne muscular dystrophy (Duchenne) in patients with confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping; VYONDYS 53 for the ...

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Produced by ElevenLabs and News Over Audio (Noa) using AI narration. Listen to more stories on the Noa app. If Donald Trump were trying to lose his trade war with China, it’s hard to see what he ...