The past few months have felt different. Our community continues to grow, yet in some ways, it feels closer — more personal. We’ve gotten to know many of you more deeply. We’ve suffered immeasurable loss and grief that has brought us together. A reminder of what’s at stake. This is the context for what you’ll find in this newsletter. As a foundation, we are sharpening our focus. This means leaning into research, pursuing answers, and working to understand Danon disease in ways that will one day give all of us real options. This mission doesn’t exist without this community. |
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April 23rd EL-PFDD with the FDA |
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On April 23rd, we participated in the Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting with the FDA, partnering with organizations working in genetic cardiomyopathies to bring your experiences and perspectives directly to regulators – making the case for gene therapies and other treatments for Danon disease. A very special thank you to everyone who participated – whether by sharing your story, joining the live meeting, or completing the survey. We showed up in a meaningful way, contributing to a significant portion of responses relative to the size of our small, rare community. It means something – we are here, and our voices need to be heard. |
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| The patient survey is still open. The report will be compiled and shared more broadly later this year – so if you haven’t contributed yet, there’s still time. Complete the survey → |
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| The team at Dr. Eric Adler’s Lab at UCSD is actively looking for participants. If you live outside the San Diego area, they have options to work with you locally. For more information, contact Eren Gunes at egunes@health.ucsd.edu. (US only) |
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| Two ongoing Danon disease studies sponsored by Rocket Pharma are now known as the LAMPLIGHT Studies: LAMPLIGHT–NH (natural history) and LAMPLIGHT–2 (gene therapy). Both US and international patients are eligible. |
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| | | Research in Danon Females |
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| We are actively exploring research options specific to females diagnosed with Danon disease. We know many of you are experiencing survey and research fatigue, which is exactly why we need your input before moving forward. If you’re interested in being part of this conversation, please reach out to jenny@danonfoundation.org. |
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Introducing the Courage Fund |
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Participating in clinical research is an act of extraordinary courage. The Courage Fund is our way of acknowledging that – offering a one-time stipend of up to $250 to individuals actively participating in a qualifying research study or clinical trial. This isn’t compensation for participation. It’s a small gesture toward a moment of normalcy, because we see the whole person behind every research journey. |
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Danon Community Survey Results |
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| Thank you to everyone who filled out our survey. We’ve learned a great deal about this community, and your responses are directly shaping our planning. We promised to share the results – and to protect privacy, we’ve aggregated the information. We’re sharing findings in the Facebook support group in bite-sized pieces, and are happy to go deeper if you have questions. The survey remains open if you’d still like to contribute.
Provide your input → |
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Your Stories – Living with Danon Disease |
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Remember Nanako from our last newsletter? Her journey on navigating Danon disease with her twin sister is so powerful. She put her story out there in hopes that it might create awareness and inspire research – and has helped us feel less alone. We’d love to hear from you too – and are creating a space to tell your story, featured on our website. We know that not everyone wants to share personal details, and we’ll work with you to respect your privacy every step of the way. |
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Danon Patient Advisory Group |
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We are moving forward with launching a Danon Patient Advisory Group. Input from this group will help guide our decisions across research, partnerships, and community programs. If you’re living with Danon disease and are interested in getting involved, we’d love to hear from you. Express your interest → |
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We’ve expanded our partnerships meaningfully over the past few months. In March, we became an official member of Global Genes and its Global Advocacy Alliance, as well as the EveryLife Foundation’s Community Congress – both give us a seat at the table for important policy conversations in the rare disease space. We continue as an Affiliate of Global Heart Hub, actively participating in its Cardiomyopathy and Women & Heart Disease Networks. In addition to connecting with other patients and organizations around the world, through this partnership, we now have a clinical research portal on our website where you can search for Danon disease trials worldwide, in multiple languages. We also continue our work with GCAC to expand access to genetic testing. If you or someone you know has been diagnosed with cardiomyopathy, free genetic testing and counseling are available through Heartgene – it’s as simple as filling out a form and receiving a test kit. Get a free genetic test through Heartgene → |
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We’re also working on additional collaborations we hope to share in our next newsletter. |
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Danon Community Monthly Meeting – Wednesday, May 13th |
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| | Our monthly online community meetings are open to anyone connected to Danon disease — patients, caregivers, family members, and loved ones. Join us Wednesday, May 13th at 8pm Eastern to connect, listen, or ask questions. |
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Webinar: Understanding Wearables, Hear Data & the OCORO Heart Study – Wednesday, May 20th |
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Join the Genetic Cardiomyopathy Awareness Consortium (GCAC) for an educational webinar on wearable devices and heart data — and an introduction to the OCORO Heart Study, a remote, observational study for people living with heart conditions. No clinic visits or procedures involved. You’ll learn who may be eligible, what participation looks like, and how to get involved. Register for the webinar → |
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Want to stay up-to-date on our latest news? Sign-up here! |
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