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Danon Disease Resources

Danon Clinical Trials Resources

FAQs about Rocket’s Gene Therapy Approach to Danon Disease

Danon Disease is 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. Danon Disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. Autophagy defects result in accumulation of proteins and other cellular debris in autophagosomes, particularly in cardiac muscle and other tissues. The only available treatment option for Danon Disease is cardiac transplantation, which is associated with substantial complications and is not considered curative.

Patients with Danon Disease receive a single (one-time) intravenous infusion with an engineered virus containing the non-mutated version of the LAMP2B gene, in an effort to restore cardiomyocyte LAMP2B function.

Eligible subjects include males aged 8 or above with 1) a pathogenic or likely pathogenic variant of the LAMP2 gene, 2) left ventricular hypertrophy with preserved systolic function (LVEF ≥50%), 3) New York Heart Association (NYHA) Class II to III, 4) hsTnI ≥20% above the ULN, and 5) ability to comply with study procedures including investigational therapy and follow-up evaluations.

Ineligible subjects include those with 1) anti-AAV9 neutralizing antibody titer >1:40, 2) intravenous inotropic, vasodilator, or diuretic therapy within the 30 days prior to enrollment, 3) presence or requirement for mechanical circulatory support (MCS), 4) presence or requirement for mechanical ventilation, 5) history of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina, and 6) prior cardiac or other organ transplantation.

Please contact the enrolling physician and their research team to learn more about eligibility.

Click here to view sites for the Phase 2 study of RP-A501 and Danon natural history trial.

Travel arrangements, hotel accommodations for patients and a family member, and daily meals / incidentals allowance for the initial evaluation, treatment, and follow-up visits will be provided.

For additional resources, we encourage you to reach out to the groups below.

If your group would like to be included in our list of patient resources, please reach out to us at
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