ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY explained in plain language.
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Rare disease drug safety tracked in tiny french study
Disease control CompletedThis study followed 3 people with acid sphingomyelinase deficiency (ASMD) who had already completed earlier studies. They received enzyme replacement therapy with olipudase alfa every 2 weeks to check for side effects and safety. The study lasted until the drug was approved for u…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 07, 2026 18:42 UTC
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Simple blood test uncovers rare diseases behind mysterious enlarged spleens
Knowledge-focused CompletedThis study tested a simple blood spot test to find two rare genetic diseases (Gaucher and ASMD) in 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. The goal was to see how common these diseases are in this group. The test measure…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 17, 2026 10:38 UTC
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Hidden causes of big spleens in kids revealed
Knowledge-focused CompletedThis study checked 60 children under 18 with an enlarged spleen that had no clear cause. Doctors looked for Gaucher disease and other rare conditions using blood tests and genetics. The goal was to find how common these diseases are in such cases.
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 12, 2026 13:40 UTC