When you're diagnosing a child with Duchenne muscular dystrophy, says Alexandra Bonner, MD, of Cleveland Clinic, it's important to not only determine if she's a carrier of the gene mutation that ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

A clinical trial testing Duchenne gene-editing therapy candidate PBGENE-DMD will soon launch in the U.S., having won ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

DMD gene therapy faces challenges in safety, efficacy, and long-term outcomes, despite FDA approval of delandistrogene moxeparvovec. Future strategies focus on novel AAV capsids, dual/triple vector ...

A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at ...

A new gene therapy treatment for Duchenne muscular dystrophy shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in the ...

The Muscular Dystrophy Association (MDA) today announced Łukasz Sznajder, PhD, MSc, Assistant Professor, Department of Chemistry and Biochemistry, University of Nevada, Las Vegas (UNLV), as the first ...

COLUMBUS, Ohio — Ohio is now the first state in the nation to begin screening newborns for Duchenne Muscular Dystrophy, Gov. Mike DeWine announced Monday. The provision was included in the state’s ...

Dosing has begun in a Phase 2 clinical trial testing the experimental oral therapy SAT-3247 in boys with Duchenne muscular ...