MPS II
Clinical trials for MPS II 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: MPS II
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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Major study tracks rare brain disorders to unlock disease secrets
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain diseases to learn how these conditions progress. Researchers will track thinking, movement, and daily living skills over time. The goal is to better understand the diseases and how treatments might affect them.
Matched conditions: MPS II
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 13, 2026 16:00 UTC