Danon disease natural history study
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This study follows people with Danon disease over time to better understand how the condition progresses. It may help guide future treatments.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child must have a confirmed genetic change (mutation) in the LAMP2 gene, which causes Danon disease.
- You must be at least 8 years old.
- If you are female, you must have a thickened heart muscle (left ventricular hypertrophy) either now or in the past.
- You cannot have had gene therapy or be in another experimental drug trial.
- If you are female, you must be 51 years old or younger.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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