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 genetic disease
Knowledge-focused Recruiting nowThis study follows up to 10 children under 2 years old with acid sphingomyelinase deficiency (ASMD) who are already receiving olipudase alfa (Xenpozyme®) as part of their routine care. Researchers will monitor side effects and immune responses over 1 to 3 years. No new treatment …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Sanofi • Aim: Knowledge-focused
Last updated Jun 23, 2026 11:54 UTC
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Simple blood test may uncover rare diseases in myeloma patients
Knowledge-focused Recruiting nowThis study aims to find out how many people with an enlarged spleen or certain blood disorders (like multiple myeloma) also have Gaucher disease or acid sphingomyelinase deficiency (ASMD). Researchers will use a simple blood spot test to check for these rare conditions. The goal …
Matched conditions: ACID SPHINGOMYELINASE DEFICIENCY
Sponsor: Fundación Española de Hematología y Hemoterapía • Aim: Knowledge-focused
Last updated Jun 15, 2026 18:56 UTC