NIEMANN-PICK DISEASES
Clinical trials for NIEMANN-PICK DISEASES explained in plain language.
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Tracking a lifesaving Treatment's safety in the tiniest patients
Disease control Recruiting nowThis study aims to monitor the long-term safety and how the body's immune system responds to olipudase alfa, an enzyme replacement therapy, in children under 2 years old with acid sphingomyelinase deficiency (ASMD), a rare and serious genetic disorder. It will follow up to 10 chi…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: Sanofi • Aim: Disease control
Last updated Mar 16, 2026 15:26 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: NIEMANN-PICK DISEASES
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC