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.

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

The Buckinghamshire man went to see his local GP. Ryan went from blood tests to muscle biopsies to DNA tests, trying to get ...

Edgewise Therapeutics Inc. (NASDAQ:EWTX) is one of the best up and coming stocks to invest in now. On June 26, Edgewise ...

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

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

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® ...

Pompe's disease is a lysosomal storage disorder caused by a genetic deficiency of GAA. The disease is associated with severe ...

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

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, ...