A mouse model study of Precision BioSciences’ gene-editing therapy PBGENE-DMD found it produced long-term functional ...

The company refused the FDA’s request and will continue shipping its therapy, Elevidys, to Duchenne patients who can still ...

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported ...

A new pioneering gene therapy Elevidys works by replacing the Duchenne variant of dystrophin with man-made version based on a version found in a patient with a milder form of the disease, called ...

Karson was at CHKD to receive a newly approved gene therapy for Duchenne muscular dystrophy, which he was diagnosed with at 20 months of age. Duchenne is a devastating genetic illness that causes ...

The study aims to evaluate the safety and efficacy of a gene therapy, PF-06939926, in boys with Duchenne Muscular Dystrophy (DMD), a severe genetic disorder characterized by progressive muscle ...

The global market for Duchenne Muscular Dystrophy Drugs, valued at US$2 billion in 2022, is projected to grow to US$27.4 billion by 2030, with a remarkable compound annual growth rate (CAGR) of 39 ...

FILE - A sign for the Food and Drug Administration is displayed outside their offices in Silver Spring, Md., on Dec. 10, 2020. The first gene therapy for Duchenne muscular dystrophy received ...