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 early-stage study tests whether giving enzyme replacement therapy to fetuses with certain genetic disorders (like MPS and Pompe disease) is safe and feasible. About 10 pregnant participants will receive the drug through the umbilical vein. The goal is to see if this approach…
Matched conditions: WOLMAN DISEASE
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 09:15 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects health information from up to 250 people with lysosomal storage diseases, including pregnant women whose fetus has a diagnosis. Researchers will track symptoms, lab results, and development over time to better understand how these rare conditions progress. The…
Matched conditions: WOLMAN DISEASE
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 26, 2026 09:02 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 patients with LAL deficiency, a rare genetic disorder. The goal is to better understand how the disease progresses and varies among people. Up to 300 patients will participate, and the data will help improve future care and …
Matched conditions: WOLMAN DISEASE
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated May 22, 2026 13:52 UTC