ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY explained in plain language.
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Tiny study tracks safety of enzyme therapy for rare disease
Disease control CompletedThis study followed 3 people with acid sphingomyelinase deficiency (ASMD) who had already completed earlier trials of olipudase alfa. They received the enzyme replacement therapy every 2 weeks to see if it remained safe and tolerable over the long term. The study ended when the d…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 22, 2026 11:58 UTC
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Blood test uncovers rare diseases in patients with mysterious enlarged spleen
Knowledge-focused CompletedThis study looked for two rare genetic diseases, Gaucher disease and acid sphingomyelinase deficiency (ASMD), in 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. Researchers used a simple blood spot test to check enzyme activity …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated Jun 18, 2026 11:50 UTC
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Rare disease hunt: enlarged spleen study in kids
Knowledge-focused CompletedThis study aimed to find out how often Gaucher disease is the cause of an unexplained enlarged spleen in children under 18. Researchers checked 60 children after ruling out common causes like infections or blood cancers. The goal was to better understand the role of rare diseases…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated Jun 15, 2026 19:02 UTC