The Accountability Crisis in Duchenne Muscular Dystrophy (DMD) Associations: Why Donations Must Lead to Measurable Outcomes
Where do DMD associations spend the donations they receive, and how much of it truly supports scientific research and clinical trials? Are donations being...
The Systemic Failure of DMD Associations: Why Duchenne Patients Are Still Waiting
Duchenne muscular dystrophy (DMD) is not a newly discovered condition, nor is it an unsolvable one. Scientific progress over the past two decades has...
NSAA Alone Is Not Enough: Why Creatine Kinase (CK), AST, ALT, and Dystrophin Levels Should Be Disclosed
So why do many DMD clinical trials avoid clearly reporting these key biomarkers? This article examines why CK, AST, ALT, and dystrophin data matter—and...
Genethon Confirms Two-Year Efficacy of GNT0004 Gene Therapy with Improvements in Creatine Kinase (CK) Levels and NSAA Scores
Gene therapy research for Duchenne muscular dystrophy (DMD) continues to advance. At the MDA Clinical & Scientific Conference in Orlando, the French gene therapy...
Compared to Its Million-Dollar Price Tag, How Much Does Elevidys Gene Therapy Improve the Condition of Children With DMD?
Elevidys, the only gene therapy approved by the FDA for Duchenne muscular dystrophy (DMD), has disappointed DMD communities and investors following the release of...