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 ...

Mutations in the dystrophin gene 1 (DMD) and in genes encoding several dystrophin-associated proteins result in Duchenne and other forms of muscular dystrophy 2. α-Dystroglycan (Dg) binds to ...

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

Pat Cornell rolls through Portland, ME on his record-breaking 125,000-mile motorcycle journey—raising critical awareness for myotonic dystrophy (DM), a progressive, inherited disease affecting more ...

Scientists have uncovered how a protein helps build and maintain vital brain connections, providing insights into the ...

The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it,' said ...