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 ...

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 ...

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

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).

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

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

The one-time treatment is approved for children with a genetic variant of Duchenne’s muscular dystrophy, which causes weakness, loss of mobility and early death in males.

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