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...
Six DMD Researches You Should Follow up in 2026
Despite significant advancements over the past ten years for DMD patients, there is still a significant unmet demand. In order to meet the need,...
‘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...
Creatine Kinase (CK), Dystrophin Level and NSAA Score in Elevidys Gene Therapy
It remains a matter of curiosity how much Creatine Kinase (CK) levels decrease, NSAA score and normal dystrophin levels increase in children with DMD...
Why Does the FDA Approve DMD Therapies That Fail in Clinical Trials?
Do gene therapies, marketed for millions of dollars, and exon-skipping therapies administered monthly and sold for thousands of dollars, actually work well? Families campaigning...