First report of systemic delivery of micro-dystrophin gene therapy in children with DMD

Researchers from Nationwide Children’s Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) — and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.