Gaucher disease, type 2
Clinical trials for Gaucher disease, type 2 explained in plain language.
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Groundbreaking trial aims to treat rare diseases in the womb
Disease control Recruiting nowThis study tests whether giving enzyme replacement therapy to fetuses with certain rare genetic diseases (like MPS I, Gaucher, or Pompe) before birth is safe and feasible. About 10 pregnant participants will receive the treatment through the umbilical vein. The goal is to see if …
Matched conditions: GAUCHER DISEASE, TYPE 2
Phase 1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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Can we outsmart Gaucher's hidden toll on lungs, bones, and brain?
Knowledge-focused Recruiting nowThis study follows 30 people with Gaucher disease who are receiving standard medical care to better understand how the condition affects the lungs, bones, and nervous system over time. Researchers will track changes in breathing, bone density, and neurological signs, along with b…
Matched conditions: GAUCHER DISEASE, TYPE 2
Sponsor: Yale University • Aim: Knowledge-focused
Last updated Aug 13, 2026 00:00 UTC
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Cough medicine repurposed: ambroxol registry launches for rare brain diseases
Knowledge-focused Recruiting nowThis study creates a registry to collect real-world information on the safety and effectiveness of ambroxol, a common cough medicine, when used at higher doses for Gaucher disease or GBA-related Parkinson disease. Researchers aim to gather data from 300 patients worldwide who are…
Matched conditions: GAUCHER DISEASE, TYPE 2
Sponsor: Shaare Zedek Medical Center • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:24 UTC