Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

The parents of a young Newry boy living with Duchenne Muscular Dystrophy (DMD) are calling for immediate action from ...

Top-line Phase 3 trial data show ambulatory boys with Duchenne continued with motor improvements two years after a single ...

A seven-year-old boy from Dublin is on his way to California to undergo testing after being diagnosed with a rare condition last year.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Robert Davidson is the first UK patient recruited to a new medical trial looking to support children with heart failure ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...

CEO Dr James Garner says at the time of the December 18 trial bombshell that knocked 85% off the value of Percheron shares, ...

Entrada plans to initiate clinical testing of ENTR-601-44 in Duchenne MD patients amenable to exon 44 skipping later this year.