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...
SonoThera’s RIPPLE Technology Offers Hope for Full-Length Human Dystrophin
SonoThera, a biotechnology company dedicated to treat the root causes of human diseases by developing the next generation of genetic medicines, announced on January...
‘One-Time’ Gene Therapies or Exon-Skipping Therapies: Which May Be Better for Duchenne’s Disease Today?
Exon skipping is a leading therapeutic approach for Duchenne muscular dystrophy (DMD), aprogressive muscle wasting disorder caused by pathogenic variants in the DMD gene...