How velmanase alfa works in children under 3 with alpha-mannosidosis
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study looks at how the drug velmanase alfa (Lamzede) works in the body of young children with alpha-mannosidosis. It's for children who started the drug before age 3.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must have a confirmed diagnosis of alpha-mannosidosis.
- Your child must have started treatment with velmanase alfa (Lamzede) before reaching 3 years old.
- Your child must have had a blood test for the disease marker before starting treatment and at least one test after 6 weeks of treatment.
- Your child cannot have had a stem cell transplant or any other experimental therapy for alpha-mannosidosis.
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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