NIEMANN-PICK DISEASES
Clinical trials for NIEMANN-PICK DISEASES explained in plain language.
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New study tracks safety of enzyme therapy in babies with rare disease
Knowledge-focused Recruiting nowThis study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are receiving olipudase alfa as part of their routine care. Researchers will monitor side effects and the body's immune response to the drug over 1 to 3 years. The goal is to g…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 01, 2026 16:01 UTC
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Scientists launch massive effort to track rare brain disorders
Knowledge-focused Recruiting nowThis study follows 1500 people with rare genetic brain diseases to learn how these conditions progress over time. Researchers measure thinking, movement, and daily living skills, and look for markers in blood and brain scans. The goal is to better understand these disorders and h…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 01, 2026 15:59 UTC