NIEMANN-PICK DISEASES
Clinical trials for NIEMANN-PICK DISEASES explained in plain language.
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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 17, 2026 04:32 UTC
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Major study tracks rare brain disorders to unlock disease secrets
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain diseases to learn how these conditions progress. Researchers will track thinking, movement, and daily living skills over time. The goal is to better understand the diseases and how treatments might affect them.
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC