Illuminating new possibilities for Danon Disease
The Need for New Danon Research
Danon Disease is caused by changes in the LAMP2 gene, which prevent cells from clearing waste properly. Over time, this buildup harms the heart and other organs, leading to serious and sometimes life-threatening complications.
Because there are no approved treatments that can slow or stop this process, new research is urgently needed to better understand the disease and develop effective therapies.
Taking part in research studies helps move these treatments forward and provides families with access to specialists who understand Danon Disease.
Our Danon Research Studies
Observational study to collect the medical history and day-to-day experiences of people with Danon Disease to push research forward.
Interventional study that helps researchers study a potential gene therapy to replace the damaged LAMP2 gene in Danon Disease.
Danon Natural History Study
Who Can Participate
Diagnosed with Danon Disease, confirmed by a Genetic Test
Those willing to provide access to medical records/history
Because Danon Disease is such a rare condition, there is little information for researchers to study to help them understand the disease and how it may be treated. Researchers need to learn from the real-life experience of people living with Danon Disease, and by joining the LAMP 2 hiStory Study, you can provide the information needed.
The goal of this study is to collect data of the signs and symptoms, key events, and the impact of the disease on quality of life, as it is managed with current treatments.
Participation does not add any new therapy, only access to your medical history and collecting your feedback through surveys and interviews with the study team.
Biological Males 8+
Biological Females 8-35
Danon Gene Therapy Study
Who Can Participate
Diagnosed with Danon Disease,
confirmed by a Genetic Test
Those with a NY Heart Association Class II or III
Gene therapy is being developed for Danon disease, with the investigational therapy designed to deliver a functional LAMP2 gene into a patient's cells to replace the damaged gene.
The goal of this clinical trial is to assess its long-term safety and effectiveness in improving heart function and other symptoms.
How is the gene therapy supposed to work?
Replacing the gene: Danon disease is caused by a mutation in the LAMP2 gene, which prevents cells from properly recycling waste. The investigationalgene therapy delivers a working copy of the LAMP2 gene into heart cells, so the cells will be able to clear waste properly.
Single-dose infusion: Treatment is given once, as a single intravenous (IV) infusion.
To download a fact sheet about the Gene Therapy Clinical Study click here.
Biological
Males 8+
Frequently Asked Questions
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A genetic test, ordered by a geneticist or another specialist, can detect whether there is a damaged or missing LAMP 2 gene.
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Collecting data about a rare disease like Danon Disease is necessary to understand how it starts, its cause, and how it progresses, in order to be able to develop a treatment for it
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Fill out and submit the form and if you match the study requirements you will be contacted by the support team who will help you connect with the study site team.
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Yes, if the person you care for starts off in the natural history study, and is eligible and willing to participate in the gene therapy study, you can apply for a prescreening interview with the Re:Start Study team who will decide if you are eligible to participate in the gene therapy study.
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The costs of the therapy and visits will be covered by the study sponsor, as is travel to the study sites.
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Participation is completely up to you and your family. You can withdraw permission to access records from the Natural History study, and discontinue the process of screening and testing for the gene therapy without any problem to you.
Ready to Participate?
Fill out the form here to contact us about joining our Lamp Light Danon Disease studies