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, ...

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 ...

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

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

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.

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

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

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

Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).