Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a ...

Satellos said that it will be advancing its DMD small molecule to a Phase II trial, following the Phase I data.

Pediatric neurologist Omer Abdul Hamid is one of the few doctors in the US who administer gene therapy for Duchenne muscular ...

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.

Multidisciplinary coordination is crucial for effective gene therapy administration in Duchenne muscular dystrophy, focusing on institutional readiness and patient-centered care. Guidelines ...

More information: Dariusz C. Górecki et al, Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy (2025). DOI: 10.1038/s41434-025-00531-y ...

Other types of gene therapies that use CRISPR-Cas9 cut DNA in a specific location and correct ... link of a gene therapy for Duchenne muscular dystrophy after a 16-year-old boy treated with ...

After a patient taking the Duchenne muscular dystrophy gene therapy Elevydis died of liver injury, Sarepta will update the label to reflect the safety signal. A patient taking Sarepta Therapeutics’ ...

It’s caused by mutations in the DMD gene, which carries the instructions for making the protein dystrophin. Approximately 70% to 80% of people with DMD inherit a genetic mutation that causes the ...

What Causes It? An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains the structure of muscle cells. People with DMD have ...

Treatment of Duchenne muscular dystrophy (DMD) was carried out for the first time Abu Dhabi: Department of Health — Abu Dhabi (DoH) has provided revolutionary gene transfer therapy for Duchenne ...