Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

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

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...

Muscular dystrophy is caused by defects in certain genes, with type determined by the abnormal gene. In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne ...

Learn More Genetic Causes of Muscular Dystrophy Is muscular dystrophy fatal? Some types of muscular dystrophy can be fatal. Certain types, including Duchenne muscular dystrophy, can lead to problems ...

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 ...