Edgewise Therapeutics, Inc., a leading muscle disease biopharmaceutical company, unveiled positive results in its sevasemten programme for Becker and Duchenne muscular dystrophies. The company ...

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

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

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

Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...

The U.S. Food and Drug Administration is due to review data on deramiocel, a cell therapy up for approval for heart disease ...

Skeletal muscle disease progression continues to slow with extended treatment (PUL v2.0) ...

I am upgrading Dyne Therapeutics stock to a 'Buy' rating due to promising late-stage DM1 and DMD drug candidates and ...

For the second time, a person with DMD has died of acute liver failure after being treated with the one-time gene therapy Elevidys.

U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s ...

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