MPS IVA
Clinical trials for MPS IVA 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 IVA
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 09:48 UTC
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New study aims to track rare bone disease without surgery
Knowledge-focused Recruiting nowThis study is for people with Morquio A, a rare disease that affects bones and breathing. Researchers will use safe, non-invasive tests like X-rays, MRI, and breathing tests to track how the disease changes over time. The goal is to better understand the disease and find better w…
Matched conditions: MPS IVA
Sponsor: Nemours Children's Clinic • Aim: Knowledge-focused
Last updated May 22, 2026 14:01 UTC
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New study tracks heart changes in rare MPS disorders
Knowledge-focused Recruiting nowThis study looks at how heart and blood vessel problems develop in people with MPS types I and IVA. Researchers will use yearly ultrasounds, blood draws, and urine tests for 4 years to track changes and find markers that could guide future treatments. The study involves 240 parti…
Matched conditions: MPS IVA
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated May 18, 2026 12:09 UTC