WOLMAN DISEASE
Clinical trials for WOLMAN DISEASE explained in plain language.
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Groundbreaking trial aims to treat fatal diseases in the womb
Disease control Recruiting nowThis study tests whether giving enzyme replacement therapy to fetuses with certain lysosomal storage diseases (like MPS disorders, Gaucher, or Pompe) before birth is safe and possible. About 10 pregnant participants will receive the drug through the fetal umbilical vein. The goal…
Matched conditions: WOLMAN DISEASE
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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New registry aims to unlock secrets of rare lysosomal diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 people with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to learn how these conditions develop and how they appear before birth. Researchers will review medical records and test samples t…
Matched conditions: WOLMAN DISEASE
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 17, 2026 13:55 UTC
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New registry aims to unlock secrets of rare LAL deficiency
Knowledge-focused Recruiting nowThis study is a registry that collects information from people with LAL deficiency, a rare genetic disorder. It does not provide any treatment but aims to better understand how the disease progresses and varies among patients. Up to 300 participants will be followed over time to …
Matched conditions: WOLMAN DISEASE
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 11, 2026 20:48 UTC