Finally, their physical therapist recommended creatine kinase ... chief research officer of the Muscular Dystrophy Association. Elevidys works by delivering a shortened dystrophin gene, called ...

Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy ... measures of physical ability, although ...

Elevidys is the gene therapy product for Duchenne muscular dystrophy (DMD) and is designed to address the underlying cause of Duchenne through targeted skeletal, respiratory and cardiac muscle ...

For patients diagnosed with ALS and other neuromuscular conditions, specialized care is critical, but often out of reach.

Italfarmaco’s oral HDAC inhibitor givinostat has been approved in the US as a treatment for Duchenne muscular dystrophy (DMD ... also saw a slower decline in physical function over 18 months ...

A new business specializing in pediatric occupational, physical, and speech therapy will be holding its grand opening later ...

Scientists were able to create a bespoke treatment for KJ Muldoon’s rare genetic disorder within six months. It could be a ...

European Commission approves Roche’s Evrysdi tablet to treat spinal muscular atrophy: Basel Thursday, June 5, 2025, 09:00 Hrs [IST] Roche announced that the European Commission ...

Nom Nom, the vet-developed, fresh take on dog food dedicated to making mealtime magical, is teaming up with Tony and Emmy Award-winning actress, singer, and proud dog parent Kristin Chenoweth to debut ...

Columnist Betty Vertin acknowledges the mixed feelings of fear and pride as her youngest son with DMD begins with high school band.