MPS I
Clinical trials for MPS I 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: MPS I
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Scientists launch major effort to map progression of devastating rare brain diseases
Knowledge-focused Recruiting nowThis study aims to understand how over 25 rare genetic disorders that damage the brain progress over time. Researchers will follow 1,500 participants, assessing their development and brain health regularly for years. The goal is to gather detailed information to help future resea…
Matched conditions: MPS I
Sponsor: University of Pittsburgh • Aim: Knowledge-focused
Last updated Mar 27, 2026 12:39 UTC
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Scientists track hidden heart risks in rare genetic disease
Knowledge-focused Recruiting nowThis study aims to understand how heart and blood vessel problems develop in people with Mucopolysaccharidosis (MPS), a rare genetic disorder. Researchers will follow 240 participants (both with MPS and healthy matches) for four years, using yearly heart ultrasounds, blood tests,…
Matched conditions: MPS I
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Mar 10, 2026 12:53 UTC