ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY 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: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Disease control
Last updated Mar 16, 2026 15:26 UTC
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Mystery spleen cases: could a hidden genetic disease be the cause?
Diagnosis Recruiting nowThis study aims to find out how often two rare genetic diseases, Gaucher disease and ASMD, occur in people with an enlarged spleen or certain blood cell cancers. Researchers will test blood samples from 210 adult patients who have these conditions but no clear cause. The goal is …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Diagnosis
Last updated Mar 25, 2026 14:08 UTC