Regulatory and clinical history around AAV microdystrophin therapies continues to shape the Duchenne muscular dystrophy (DMD) landscape after the FDA granted accelerated approval to delandistrogene moxeparvovec‑rokl (Elevidys) in 2023. Recent reviews and program updates highlight ongoing efforts to optimize vector design, durability, and safety monitoring in both ambulatory and non‑ambulatory patients, including microdystrophin expression assays and immune management. Ongoing post‑approval studies and newly reported preclinical advances aim to address durability and patient selection, underscoring how approved AAV therapies are setting standards and regulatory expectations for future systemic gene‑replacement programs.
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