The estimated cost that Belfast Trust has produced seems high and does not align with any other figures we have seen from ...

Therapeutics provided a safety update regarding Elevidys, an FDA approved gene therapy for patients with Duchenne muscular ...

Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for ...

Sarepta said it was halting shipments of its Duchenne gene therapy for patients who can no longer walk, following the death ...

BASEL, Switzerland I June 15, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new dosing restrictions, effective immediately, for ELEVIDYS™ ...

Sarepta Therapeutics on Sunday said there had been a second reported case of acute liver failure resulting in death after a patient received the company's gene therapy for a rare form of muscular ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety ...

It's a rare genetic disorder affecting less than one percent of the population, and three-year-old Ari Knowles has it.

Eric, a beloved brother and cousin, peacefully passed away on May 25, 2025, after bravely facing the challenges of Duchenne ...

A tractor run in Keld, Swaledale has raised £5,500 for Duchenne UK, supporting research into Duchenne muscular dystrophy.

A Horsham family is battling to get hold of a drug that will slow the progress of their son's muscular dystrophy.

A tractor run in Keld, Swaledale has raised £5,500 for Duchenne UK, supporting research into Duchenne muscular dystrophy.