Real-world experience with gene therapy in Duchenne muscular dystrophy center readiness and patients safety: report from Qatar.

ABSTRACT: Duchenne Muscular Dystrophy is a rare, X-linked neuromuscular disorder that leads to progressive muscle degeneration, loss of ambulation, and premature mortality due to respiratory and cardiac failure. Historically, Duchennke Muscular Dystrophy has been managed through supportive and symptomatic treatments, with limited options for disease modification. However, advancements in gene therapy have introduced promising interventions aimed at addressing the underlying dystrophin deficiency. Delandistrogene moxeparvovec (Elevidys) received accelerated approval from the U.S. Food and Drug Administration in June 2023 for ambulatory children aged 4-5 years with a confirmed diagnosis of Duchenne Muscular Dystrophy. This approval represented an advancement, offering a disease-modifying therapy at an

SUBMITTER: Osman MF 

PROVIDER: S-EPMC12932109 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature