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 trials of the enzyme replacement therapy olipudase alfa. The goal was to keep an eye on safety and side effects while providing the drug until it was officially covered in …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 17, 2026 02:55 UTC
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Simple blood spot test uncovers rare diseases behind mysterious spleen swelling
Knowledge-focused CompletedThis study looked at 122 adults with an unexplained enlarged spleen or who had their spleen removed for unknown reasons. Researchers used a dried blood spot test to check for two rare genetic disorders: Gaucher disease and acid sphingomyelinase deficiency (ASMD). The goal was to …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated Jun 15, 2026 19:04 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