Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...

Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the ...

REGENXBIO (RGNX) reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE trial of ...

Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that ...

Capricor Therapeutics (CAPR) released expectation-beating results for the fourth quarter, sending the shares up over 5% in aftermarket trading.

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...