ACID SPHINGOMYELINASE DEFICIENCY
Clinical trials for ACID SPHINGOMYELINASE DEFICIENCY explained in plain language.
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New study tracks safety of enzyme therapy in babies with rare disease
Knowledge-focused Recruiting nowThis study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are receiving olipudase alfa as part of their routine care. Researchers will monitor side effects and the body's immune response to the drug over 1 to 3 years. The goal is to g…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated May 01, 2026 16:01 UTC
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Hidden genetic diseases may lurk in myeloma patients
Knowledge-focused Recruiting nowThis study aims to find out how common Gaucher disease and acid sphingomyelinase deficiency (ASMD) are in people with monoclonal gammopathies or multiple myeloma. Researchers will test blood samples from 210 adults who have an enlarged spleen or low platelets. The goal is to impr…
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated May 01, 2026 15:57 UTC