Clinical Trials:

Danon Disease

Danon Clinical Trials Resources

RP-A501 is being developed for Danon Disease, a rare, genetic disorder characterized by thickening and weakening of the heart muscle, often resulting in heart failure, and for male patients, frequent death during adolescence or early adulthood.

A non-randomized, open-label Phase 1 trial of RP-A501 aims to enroll both pediatric and young adult male patients in escalating dose cohorts. Following the review of safety data from the first cohort, all subsequent cohorts will include 2-4 patients per cohort. The study is designed to assess the safety and tolerability of a single intravenous (IV) infusion of RP-A501.

For more details about the trial, visit

For more information about Danon Disease, visit our Patients and Community page or email us at

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