Diagnosed with Danon Disease?
Clinical studies can shed new light on the progression of the disease and investigate new potential treatments.
Join a Clinical Study in Danon Disease
Danon disease is caused by changes in the LAMP2 gene, which prevents cells from clearing waste properly. Over time, waste buildup can harm the heart and other organs, leading to decreased heart function and other serious complications.
Doctors currently treat this disease by managing its symptoms, focusing on heart problems, since they are the most dangerous. While a heart transplant is currently the only intervention that can restore the heart's function, it is difficult to get because of a shortage of donor hearts and the risk of serious side effects. Scientists are currently studying other investigational treatment options.
Joining a Danon disease clinical study provides the opportunity to contribute to research on investigational new treatments led by expert Danon disease clinicians.
Danon Disease Natural History Study
You have the opportunity to help researchers better understand Danon disease and make an impact on the care of future patients. Because Danon disease is such a rare condition, and there is limited information available, this study helps researchers understand the disease and how it may potentially be treated. Researchers need to learn from the real-life experiences of people living with Danon disease, and by joining the LAMPLIGHT Natural History Study, you can provide the information needed.
What to expect during the study:
Yearly lab and echocardiogram (ECHO) and electrocardiogram (EKG) assessments, reporting on key events, and the impact of the disease on quality of life, as it is managed with current treatments, for a total of 3 years.
You do not receive any new therapy as part of the study.
Visits take place at a study site or, in some cases, can be conducted remotely.
You will continue to see your doctor for regular care.
Note: Costs associated with participation are covered by the study.
Who Can Participate?
Males, age 8 years or older
Females, age 8 to 50 years
Diagnosed with
Danon disease
The LAMPLIGHT Studies have study sites in the USA and internationally. For a full list of study site locations click here.
Frequently Asked Questions
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A genetic test, ordered by a healthcare provider, can detect whether there is a damaged or missing LAMP2 gene.
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A natural history study is also called an “observational study” because unlike an interventional study, patients do not receive any treatment. Researchers observe and collect information, including lab assessments, about a condition. People in the study stay on the treatments they already use and will continue to see their doctor for regular care.
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First, fill out and submit the form with the basic information. The support team will then contact you to review the information and discuss the study’s requirements. If the patient meets the criteria, the support team will connect you with a clinical study site.
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Yes. If a male patient joins the Natural History study and is interested in learning more about participation in the Gene Therapy study, a discussion should be had with the Natural History study Principal Investigator to receive a referral to the Gene Therapy study team.
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The costs associated with study visits and treatment, including travel, lodging, and meals are covered by the study.
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Participation is voluntary, and up to you and your family. You can withdraw from the Natural History study, and discontinue the process of screening and testing for the Gene Therapy study at any time. This will not impact your regular clinical care.
Sources:
1. https://clinicaltrials.gov/study/NCT06092034 and https://clinicaltrials.gov/study/NCT06214507
2. Data on file, Rocket Pharmaceuticals, Inc. 2026.
3. https://pmc.ncbi.nlm.nih.gov/articles/PMC4133537/#sec7
4. Clinical Profile of Cardiac Involvement in Danon Disease | Circulation: Genomic and Precision Medicine https://pubmed.ncbi.nlm.nih.gov/34775111/
5. Boucek D, Jirikowic J, Taylor M. Natural history of Danon disease. Genet Med. 2011;13(6): 563-568.
6. Brambatti M, Caspi O, Maolo A, et al. Danon disease: Gender differences in presentation and outcomes. Int J Cardiol. 2019;286:92-98.
Ready to Join?
Fill out the form here so we can contact you with more information about joining our LAMPLIGHT Danon disease studies