ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY explained in plain language.
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Hidden genetic diseases may be common in blood cancer patients, new study suggests
Knowledge-focused Recruiting nowThis study aims to find out how often Gaucher disease and acid sphingomyelinase deficiency (ASMD) occur in people with enlarged spleens or certain blood disorders like multiple myeloma. Researchers will use a simple blood test to check for these rare genetic conditions. The goal …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 20, 2026 11:53 UTC
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Researchers track safety of rare disease drug in babies
Knowledge-focused Recruiting nowThis study follows up to 10 children under 2 years old who are already receiving olipudase alfa for acid sphingomyelinase deficiency (ASMD). The goal is to monitor long-term safety and whether the body develops antibodies against the drug. No new treatment is given; researchers s…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 18, 2026 12:09 UTC