MPS IVA
Clinical trials for MPS IVA 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 IVA
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Mar 30, 2026 14:32 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 IVA
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Mar 10, 2026 12:53 UTC