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 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 IVA
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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New study aims to unlock secrets of 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 want to learn more about how the disease progresses by using safe, non-invasive tests like X-rays, MRIs, and breathing tests. The goal is to find better ways to measure the disea…
Matched conditions: MPS IVA
Sponsor: Nemours Children's Clinic • Aim: Knowledge-focused
Last updated May 17, 2026 03:11 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 17, 2026 03:10 UTC