News

MedPage Today on MSN1d
Neurodevelopmental and Psychiatric Disorders in Duchenne Muscular Dystrophy
Dystrophin's isoforms are thought to contribute variably to neuropsychiatric disorders, behavioral disorders, and ...
MedPage Today on MSN1d
Cardiac Complications of Duchenne Muscular Dystrophy
Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the ...
The Brighterside of News on MSN42m
Antimalarial drug could treat cystic fibrosis, Duchenne muscular dystrophy, as well as some cancers
In many genetic diseases, a small error in the DNA code can have devastating effects. These errors, called nonsense mutations ...
National Hero John Davidson Hits the Road Once Again to Honour His Son and Push for a Cure for Duchenne Muscular Dystrophy
John Davidson and his 15-year-old son Jesse captured the hearts of Canadians when John pushed Jesse across Ontario in his ...
Entrada Therapeutics Appoints Maha Radhakrishnan, M.D., to its Board of Directors
Maha Radhakrishnan, MD Maha Radhakrishnan, MD BOSTON, June 03, 2025 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: ...
Fierce Biotech5d
PepGen ends all work on Duchenne muscular dystrophy after lead asset fails to raise dystrophin levels
Months after causing safety concerns that led the FDA to hit a related trial with a clinical hold, a phase 2 trial of ...
Fight to get free 'life-changing' drug almost over
A family say it is outrageous they have had to fight for access to a free drug that slows the progression of a muscle-wasting ...
STV News5d
Hope for families as new medication rolled out to first Scottish children
Givinostat can slow the progression of Duchenne muscular dystrophy and is now available to some patients via an early access ...
The American Journal of Managed Care4d
Hurdles Remain Despite Progress in Gene Therapy for DMD
Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...
‘It won’t stop me’: Hong Kong teen battles condition to live ‘ordinary days’
Hayden Yung finds ways to persevere with muscular dystrophy, but experts say targeted help is needed both for children and ...
PepGen Abandons Duchenne Program, Shifts Spotlight To Another Rare Disease Candidate
PepGen ends Duchenne drug program after weak Phase 2 results, pivots to DM1 treatment showing early signs of splicing ...
PepGen to discontinue development of experimental Duchenne therapy; shares drop
Drug developer PepGen said on Wednesday it will discontinue development of its experimental therapy for Duchenne muscular dystrophy (DMD) after it failed to boost production of a protein in a ...