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 ...

Three years after Elevidys treatment, boys with DMD show sustained motor function compared with expected disease progression.

UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section ...

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 ...

The FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys), the first gene therapy for Duchenne muscular dystrophy, the agency announced Thursday. The adeno-associated virus-based ...

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 ...

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 ...

Duchenne muscular dystrophy is caused by mutations in the dystrophin encoding DMD gene that disrupt the reading frame. Muscle fibers lacking dystrophin are more sensitive to damage and over time ...

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 ...