Duchenne muscular dystrophy (DMD) is the most common type. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms ...

It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which codes for the protein dystrophin. Dystrophin is a protein that plays a role in strengthening muscle fibers ...

Duchenne muscular dystrophy results from mutations in the DMD gene that encodes the dystrophin protein. There are many types of mutations that can cause the disease; each disrupts the reading frame ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Roche has bought ex-US rights to Sarepta’s Duchenne muscular dystrophy (DMD ... into the virus vector used by Sarepta to insert the DNA sequence into the host. So instead the biotech has ...

Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a view to ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Duchenne muscular dystrophy (DMD) is a rare genetic disorder that is characterised by progressive muscle degeneration and weakness, and is caused by mutations in the dystrophin gene. Currently ...

In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne muscular dystrophy. In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne ...