Study of acid sphingomyelinase deficiency in children and adults
Part of Blood & lymphatic, Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study follows people with acid sphingomyelinase deficiency (ASMD) over time to learn more about how the condition changes. You won't get any new treatment, but your participation will help doctors better understand ASMD.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You or your child are at least 2 years old.
- A doctor has confirmed the diagnosis of acid sphingomyelinase deficiency (ASMD) with a special blood test.
- You are willing to take part in the study and have not said you don't want to.
- You are covered by French social security (or have similar entitlements).
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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