Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...

The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with ...

Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...

RGX-202 shows promising efficacy in Duchenne muscular dystrophy, improving functional outcomes and biomarker expression in ...

Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...

Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...

Another person treated with Elevidys suffered acute liver failure, leading Sarepta to discontinue treatment in certain ...

The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...