A Sarepta Therapeutics gene therapy that failed its confirmatory test has now converted its accelerated FDA approval into a traditional one, expanding use of the treatment to a wider group of Duchenne ...

Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored dystrophin protein ...

Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new ...

Involvement of different brain dystrophin isoforms was assessed in 3 groups of young males diagnosed with DMD. On their first assessment, 43 of the 70 patients with DMD had normal intelligence ...

Viltolarsen targets dystrophin gene mutations, enabling functional dystrophin protein production, crucial for muscle health in patients with DMD. Consistent viltolarsen treatment slowed disease ...

Duchenne muscular dystrophy (DMD), the most lethal musculoskeletal disease, is caused by mutations in the X-linked gene dystrophin. Patients with DMD show delayed motor milestone development at 2-6 ...

Pfizer’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, did not meet its primary end point of improvement in motor function in ambulatory patients with Duchenne muscular ...

- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose Dependent 27% Mean Splicing Correction Across All Patients in the 5.4 mg/kg Cohort at 3 Months - - DYNE-101 Showed Improvement in Myotonia, ...

Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop ...