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EL-PFDD Meeting on Genetic Cardiomyopathies and Gene Therapy

Help us Present the Patient Perspective on Living with a Genetic Cardiomyopathy to the FDA

Quick Facts

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 – and why new potential therapies, such as gene therapies, are urgently needed for these conditions.

Date and time: April 23, 2026 | 12-5 pm Eastern Time

Who this meeting is for: We encourage everyone with a genetic cardiomyopathy condition – including:

• Arrhythmic Cardiomyopathy (ARVC, also known as ACM)
• Hypertrophic Cardiomyopathy (HCM)
• Dilated Cardiomyopathy (DCM)
• Amyloidosis Related Cardiomyopathy (ATTR-CM)
• Friedreich’s Ataxia Related Cardiomyopathy (FA-related HCM)
• Danon Disease (LAMP2)

How you can participate: If you have a genetic heart muscle condition, we need your input to make this meeting a success! 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 below. If you’re 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.

About the EL-PFDD for Genetic Cardiomyopathies

The SADS Foundation is pleased to announce that we will be participating in our fourth Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD) for the Food and Drug Administration (FDA), researchers, biopharma companies, healthcare providers, product developers and federal regulatory partners so they can understand the perspective of patients living with cardiomyopathies – and why new potential therapies, such as gene therapies, are urgently needed for these conditions.

This year’s EL-PFDD meeting, held in conjunction with our partners at the Hypertrophic Cardiomyopathy Association (HCMA), Amyloidosis Research Consortium (ARC), Friedreich’s Ataxia Research Alliance (FARA), Danon Foundation, and Dilated Cardiomyopathy (DCM) Foundation, will focus on genetic cardiomyopathies and the development of gene therapies on April 23, 2026.

Read more about our EL-PFDD for LQTS 👉
Read more about our EL-PFDD for CPVT 👉
Read more about our EL-PFDD for ARVC (also known as ACM) 👉

What is an Externally-Led Patient-Focused Drug Development Meeting?

Externally-Led Patient-Focused Drug Development (EL-PFDD) meetings give the Food and Drug Administration (FDA) and other key stakeholders — including researchers, biopharma companies, healthcare providers, product developers and federal regulatory partners — an important opportunity to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact the disease has on patients’ daily lives, patients’ experiences with currently available treatments, and patient’s priorities for therapeutic outcomes. These inputs may also inform FDA’s decisions and oversight both during drug development and their reviews of marketing applications for new therapies.

How is this meeting different than the previous EL-PFDD meeting for ACM?

In 2023, the SADS Foundation held an EL-PFDD meeting on ARVC (also known as ACM). This meeting will cover genetic cardiomyopathies more broadly – including Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Amyloidosis, Friedreich’s Ataxia, and Danon Disease. By bringing together many cardiomyopathy conditions, we hope to help advance research for gene therapies for the cardiomyopathy space more broadly – helping all people with genetic cardiomyopathy conditions, and their families.

This meeting will build on the previous evidence and momentum from our past three EL-PFDD meetings as we continue to educate the FDA and other stakeholders about these conditions and the urgent need for better, more targeted therapies that will solve our community’s most critical unmet needs.

What happens during the meeting?

During the meeting, the hosts and co-moderators will be looking for the cardiomyopathy community to participate in polls, send in comments, or call in live. There will be a brief 10-15 minute overview of these conditions by a clinical expert, followed by two sessions with patient panelists, callers, reading of submitted comments, and live anonymous polling.

We’d love for you to view and participate in the entire meeting, but if you can only attend part of the meeting, that would also be greatly appreciated. Every voice counts and helps the FDA and other and stakeholders understand the critical need for better treatments for cardiomyopathy conditions.

What happens after the meeting?

The comments and information shared before, during, and after the meeting will be consolidated into a Voice of the Patient report. This report will be publicly available and will be provided to the FDA and to researchers developing new therapies for cardiomyopathies.

View the Voice of the Patient Report for LQTS
View the Voice of the Patient Report for CPVT
View the Voice of the Patient Report for ACM 

FAQ (Frequently Asked Questions)

Is this meeting free? Absolutely – we want all cardiomyopathy members to participate, and there is no charge for being a part of this meeting.

Can families outside of the USA participate? Yes – the meeting is open to all genetic cardiomyopathy families across the globe.

What about families with deceased loved ones? We want – and need! – to hear and learn from you as well.

What if I have a cardiomyopathy condition, but I’m gene elusive? We still value your participation and attendance – you are not only welcome, but encouraged to be a part of this meeting.

Do I need a computer to attend? No, you can participate from your mobile device or tablet – if you can read this web page (and you are right now), you can be a part of the meeting.

What will be the primary meeting language? It will be in English.

Will the meeting be recorded? Yes – it will be available immediately after the meeting ends on this page (hosted by YouTube so everyone can easily access it).

Take the Genetic Cardiomyopathy EL-PFDD Survey
 

This brief survey should take no more than five minutes to complete. You have the option to either submit the survey with your name or anonymously.

The survey questions are intended to capture and document individuals’ experiences and lived experiences of impacted individuals and their families/caregivers. We will use this information to create our “Voice of the Patient” report to be submitted to the FDA following the EL-PFDD. You will be given the opportunity to provide contact information if you wish to receive a copy of the final report.