ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY explained in plain language.
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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 17, 2026 10:44 UTC
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Hidden genetic diseases may be behind some blood cancers, new study suggests
Knowledge-focused Recruiting nowThis study aims to find out how many people with certain blood disorders (monoclonal gammopathies or multiple myeloma) also have undiagnosed Gaucher disease or acid sphingomyelinase deficiency (ASMD). Researchers will test 210 adults who have an enlarged spleen or low platelets u…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 17, 2026 10:26 UTC