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 of the enzyme replacement therapy olipudase alfa. The goal was to monitor safety and side effects while providing the drug until it was officially covered in France…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Phase: PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 26, 2026 05:41 UTC
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Simple blood test uncovers rare diseases in patients with unexplained enlarged spleen
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 simple 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 May 26, 2026 05:31 UTC
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Hidden causes of big spleens in kids revealed
Knowledge-focused CompletedThis study checked 60 children with unexplained spleen enlargement to see how many had Gaucher disease or other rare conditions. Researchers looked at medical history and tests to find the cause. The goal was to better understand why some children have enlarged spleens without a …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 22, 2026 14:02 UTC