GAUCHER DISEASE TYPE 3
Clinical trials for GAUCHER DISEASE TYPE 3 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: GAUCHER DISEASE TYPE 3
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 11:22 UTC
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Brain chemistry clues sought in rare gaucher disease study
Knowledge-focused Recruiting nowThis study aims to identify brain chemical markers in adults with Type 3 Gaucher disease using a special MRI technique called magnetic resonance spectroscopy. Researchers will measure up to 18 brain metabolites to see if they can track disease activity and treatment response. Onl…
Matched conditions: GAUCHER DISEASE TYPE 3
Sponsor: University of Minnesota • Aim: Knowledge-focused
Last updated May 26, 2026 11:25 UTC