Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

Dyne Therapeutics (DYN) announced that the European Commission has granted orphan drug designation for DYNE-251 for the treatment of Duchenne ...

The antimalarial drug mefloquine could help treat genetic diseases such as cystic fibrosis, Duchenne muscular dystrophy, as ...

Michael Rankin, 12, who is desperately waiting for access to the drug, said: “Every second they don’t get this through, one ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.