Clinical Trials:
Danon Disease

RP-A501 for Danon Disease
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.
An open-label, global Phase 2 pivotal clinical trial of RP-A501 for the treatment of Danon Disease has commenced. The trial is expected to enroll 12 male patients from the U.S. and EU. The trial will assess a co-primary endpoint consisting of LAMP2 protein expression and left ventricular (LV) mass reduction from baseline at 12 months.
Natural History Study
For more information about Danon Disease, visit our Patients and Community page or email us at clinicaltrials@nullrocketpharma.com.
For more details about the RP-A501 for Danon Disease clinical trial, visit clinicaltrials.gov.
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