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Danon Disease Natural History Study

• Source: An Observational Study of Genetic Cardiomyopathy, Danon Disease

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A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

• Source: Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector

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Natural History of Danon Disease

• Source: A Retrospective, Multicenter, International Study to Characterize the Natural History of Danon Disease

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Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B

• Source: A Clinical Study Evaluating a Recombinant Adeno-Associated Virus Serotype 9 (rAAV9) Capsid Containing the Human Lysosome-Associated Membrane Protein 2 Isoform B (LAMP2B) Transgene (RP-A501;

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The Natural History of Danon Disease

• Authors : Rocket Pharmaceuticals Inc.; Eric Adler, MD, Professor of Medicine

• Source: The Natural History of Danon Disease

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