The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

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

Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...

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

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...

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

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

The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...

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

Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).

Duchenne muscular dystrophy families are sharing stories about Elevidys complications on social media. But they aren’t ...

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