By deleting the Mss51 gene, scientists were able to reduce fatigue, increase endurance, and ease mitochondrial impairment in a mouse…
Articles by Joana Carvalho, PhD
CureDuchenne has launched a new initiative to prioritize the needs of the Duchenne muscular dystrophy (DMD) community during the…
Throughout 2020, Muscular Dystrophy News Today brought you daily coverage of important discoveries, treatment advancements, clinical trial findings, and other…
Edasalonexent, a potential oral therapy for Duchenne muscular dystrophy (DMD), can prevent the development of heart disease…
Santhera Pharmaceuticals has decided to stop SIDEROS, its Phase 3 trial assessing the effectiveness of Puldysa (idebenone) at…
A single dose of SRP-9001, Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy…
The experimental therapy vamorolone can improve motor function significantly in boys with Duchenne muscular dystrophy (DMD), without stunting their growth and…
Sarepta Therapeutics has completed a meeting with the Office of Tissues and Advanced Therapies (OTAT) that clarified regulatory…
The National Institute for Health and Care Excellence (NICE) is urging patient organizations to gather feedback from those with…
A single administration of the gene therapy SRP-9003 at a low dose continues to lead to significant improvements in…