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 ...

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 ...

Ullrich congenital muscular dystrophy (UCMD), due to mutations in the collagen VI genes, is an autosomal recessive form of CMD, commonly associated with distal joints hyperlaxity and severe course. A ...

Duchenne Muscular Dystrophy (DMD) is a severe, X-linked genetic disorder characterized by progressive muscle degeneration affecting the skeletal, respiratory, and cardiac muscles.