Study of acid sphingomyelinase deficiency in adults and children
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study follows the natural history of ASMD, a rare enzyme deficiency. It can help researchers understand how the condition progresses in both adults and children.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 2 years old.
- You must have a confirmed diagnosis of ASMD (acid sphingomyelinase deficiency).
- You must be enrolled in social security or entitled to benefits (excluding AME).
- You must not be under guardianship, trusteeship, judicial protection, detention, or deprived of liberty.
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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