In an opinion article, investigators argue that gene therapies may not achieve their full potential if the innate inflammation in Duchenne muscular dystrophy is not effectively managed.

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

Sarepta Therapeutics Inc. misled investors about the safety and prospects for one of its Duchenne muscular dystrophy ...

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

Edgewise Therapeutics Inc. (NASDAQ: EWTX) is one of the best up and coming stocks to invest in now. On June 26, Edgewise Therapeutics announced positive results from its sevasemten program for Becker ...

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

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

Duchenne Muscular Dystrophy Market Size, Epidemiology, In-Market Drugs Sales, Pipeline Therapies, and Regional Outlook ...

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

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