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

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 ...

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 ...

Avidity Biosciences, Inc. (NASDAQ:RNA) on Wednesday shared new data from participants treated continuously with del-zota for one year in the EXPLORE44 and EXPLORE44-OLE trials for Duchenne muscular ...

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

The FDA expanded the approval of delandistrogene moxeparvovec-rokl (Elevidys) gene therapy for Duchenne muscular dystrophy on Thursday to include ambulatory or non-ambulatory patients ages 4 years and ...

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 ...

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 ...