Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in Duchenne muscular dystrophy.

Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...

Brodie Pankhurst suffers from Duchenne muscular dystrophy, which progressively weakens his muscles. The cruel illness has ...

"The journey from first symptom to diagnosis and finally treatment can be long and challenging for patients with rare ...

Muscular Dystrophy UK is the leading charity for more than 110,000 children and adults in the UK living with one of over 60 ...

Somite Therapeutics, a fully integrated TechBio company leveraging big data and AI to introduce novel cell replacement therapies, today announced that the U.S. Food and Drug Administration (FDA) has ...

To provide hope for families grappling with Duchenne muscular dystrophy across Upstate New York, Buffalo-based Suneel’s Light Foundation has launched a new community grant program, Brighter Days Commu ...

Dr. Laila Bastaki, Head of the Genetic Diseases Center at the Ministry of Health, emphasized the importance of incorporating ...

KUWAIT CITY, Sept 11: Assistant Undersecretary for Medical Support Services at the Ministry of Health, Dr. Abdullah Al-Fars, ...

The railway tunnel linking Broad Street and Hood Road in Barry was lit up in red out of respect for World Duchenne Awareness Day ...

Avidity supports World Duchenne Awareness Day, International Myotonic Dystrophy Awareness Day, FSHD Society Walk & Roll to Cure FSHD and Global Genes Week in RARE SAN DIEGO, Sept. 5, 2024 /PRNewswire/ ...