AAV9.LAMP2B gene therapy for Danon disease: from early clinical experience to evolving safety and trial design considerations

Alessia Argirò , Iacopo Olivotto

Rare Disease and Orphan Drugs Journal ›› 2026, Vol. 5 ›› Issue (3) : 30

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Rare Disease and Orphan Drugs Journal ›› 2026, Vol. 5 ›› Issue (3) :30 DOI: 10.20517/rdodj.2026.26
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AAV9.LAMP2B gene therapy for Danon disease: from early clinical experience to evolving safety and trial design considerations
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Alessia Argirò, Iacopo Olivotto. AAV9.LAMP2B gene therapy for Danon disease: from early clinical experience to evolving safety and trial design considerations. Rare Disease and Orphan Drugs Journal, 2026, 5 (3) : 30 DOI:10.20517/rdodj.2026.26

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