NIEMANN-PICK DISEASES
Clinical trials for NIEMANN-PICK DISEASES explained in plain language.
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Scientists watch rare brain diseases unfold in 1,500 patients
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 26, 2026 08:19 UTC
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Researchers track safety of rare disease drug in babies
Knowledge-focused Recruiting nowThis study follows up to 10 children under 2 years old who are already receiving olipudase alfa for acid sphingomyelinase deficiency (ASMD). The goal is to monitor long-term safety and whether the body develops antibodies against the drug. No new treatment is given; researchers s…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 18, 2026 12:09 UTC