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

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

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 ...

Participants with Duchenne muscular dystrophy (DMD) who received RGX-202 demonstrated functional improvement and better ...

The drug, Givinostat, has been approved as safe for use but Belfast Health Trust, NI's specialist centre for the condition, ...

The findings support the clinical benefits of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) ...

Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the ...

Families are now facing a postcode lottery over who gets the drug givinostat, which can slow progress of Duchenne muscular ...

U.S. FDA successfully completed Pre-License Inspection; Company expects facility will meet all requirements to support ...

The parents of a nine-year-old boy battling a devastating muscle-wasting condition say new research could offer them more ...

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of cardiac ...

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