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Help us Share the Patient Perspective on Living with Danon Disease to the FDA

Purpose: To bring the patient experience to the FDA (Food and Drug Administration), researchers, biopharma companies, healthcare providers, product developers and federal regulatory partners so they can understand the perspective of patients living with cardiomyopathies – including Danon disease – and why new potential therapies, such as gene therapies, are urgently needed for these conditions.

Date and Time: Thursday, April 23rd, 2026, 12-5pm Eastern

How to Participate: If you have a genetic cardiomyopathy condition, you can share your lived experiences with the FDA via a five-minute survey here, and we encourage you to register to attend the meeting. If you are able to attend live, you can participate in polling questions about your health experiences that will help shape the future of new treatments for your condition. More information will be available in the coming weeks to provide you with additional information on how to attend the meeting virtually.

EL-PFDD Survey

The Danon Foundation is working with the Hypertrophic Cardiomyopathy Association (HCMA) on a patient survey to help make the Food and Drug Administration (FDA) better aware of all types of cardiomyopathy and the lived patient experience. We would appreciate it if you would take this survey.

This Externally Led Patient-Focused Drug Development (EL-PFDD) initiative aims to provide a focused understanding of conditions associated with genetic cardiomyopathies, including Danon disease—its burdens, its impact on daily life, and patient perspectives on current and emerging treatments, including advanced genetically-based therapies.

The survey is anonymous and you may choose to provide your name and contact information if you would like to receive a copy of the final report, but it is not required. Participation is completely voluntary, and you may exit the survey at any time.

👉 Take the survey here:
https://www.surveymonkey.com/r/expfdd2026

Please note that while this survey is to help inform FDA, we welcome individuals globally to respond. You do not need to reside in the U.S. to complete the survey.

Thank you for helping ensure that the voices of the Danon disease community are heard.