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 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: MPS II
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 10:39 UTC
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Scientists watch rare brain diseases unfold in 1,500 patients
Knowledge-focused Recruiting nowThis study follows 1,500 people with rare genetic brain disorders like MLD, Krabbe disease, and Batten disease. Researchers track thinking, movement, and language skills over time, and collect blood and MRI scans. The goal is to learn how these diseases progress, not to test a tr…
Matched conditions: MPS II
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated May 26, 2026 10:35 UTC