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

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

Donald Trump continues to make waves in biopharma; Sage rejects Biogen’s unsolicited takeover offer; the obesity space sees ...

BOSTON - PepGen Inc. (NASDAQ: PEPG), a biotechnology firm focused on oligonucleotide therapies for severe neuromuscular and neurological diseases, is progressing with its CONNECT clinical program for ...

The data showed reduced difficulties in standing, walking and running that were statistically significant, the company said.

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

PepGen (PEPG) “provided recent updates on its CONNECT clinical program investigating PGN-EDO51 in Duchenne muscular dystrophy for patients ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

(RTTNews) - Sarepta Therapeutics, Inc. (SRPT) Monday reported positive topline results from Part 2 of the Phase 3 study, dubbed EMBARK, of its gene therapy Elevidys approved for the treatment of ...

offering new pathways for understanding and treating Duchenne Muscular Dystrophy (DMD). A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to ...

Most children with Duchenne muscular dystrophy will need a wheelchair to get around by the time they're teenagers. The disease also damages the heart and the muscles needed to breathe, which can ...

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...