WOLMAN DISEASE
Clinical trials for WOLMAN DISEASE explained in plain language.
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Doctors test Life-Saving enzyme treatment for babies before they are born
Disease control Recruiting nowThis early-stage study is testing whether giving enzyme replacement therapy to fetuses still in the womb is safe and feasible. It aims to treat rare, severe genetic disorders that often cause life-threatening complications before or shortly after birth. The hope is that treating …
Matched conditions: WOLMAN DISEASE
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Rare liver disease patients tracked in global effort to improve care
Knowledge-focused Recruiting nowThis study is creating a worldwide registry to collect long-term health information from people diagnosed with Lysosomal Acid Lipase (LAL) Deficiency, a rare genetic disorder that causes severe liver and heart problems. By tracking up to 300 patients over time, researchers aim to…
Matched conditions: WOLMAN DISEASE
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Knowledge-focused
Last updated Mar 25, 2026 14:08 UTC