Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease. Greenberg B, Taylor M, Adler E, Colan S, et al. N Engl J Med 2025;392:972-983.

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Greenberg B, Taylor M, Adler E, Colan S, et al. Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease. N Engl J Med 2025;392:972-983.
PMID: 39556016