MPS IVA
Clinical trials for MPS IVA explained in plain language.
Never miss a new study
Get alerted when new MPS IVA trials appear
Sign up with your email to follow new studies for MPS IVA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
By submitting, you agree to our Terms of use
-
Groundbreaking trial aims to treat rare diseases in the womb
Disease control Recruiting nowThis study tests whether giving enzyme replacement therapy to fetuses with certain rare genetic diseases (like MPS I, Gaucher, or Pompe) before birth is safe and feasible. About 10 pregnant participants will receive the treatment through the umbilical vein. The goal is to see if …
Matched conditions: MPS IVA
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
-
New study aims to decode rare bone disease without surgery
Knowledge-focused Recruiting nowThis study is for children with Morquio A, a rare genetic disease that affects bones and breathing. Researchers will use safe, non-invasive tests like MRI, X-rays, hearing tests, and walking analysis to track how the disease changes over time. The goal is to better understand the…
Matched conditions: MPS IVA
Sponsor: Nemours Children's Clinic • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:33 UTC
-
New study aims to uncover heart risks in rare MPS diseases
Knowledge-focused Recruiting nowThis study looks at how heart and blood vessel problems develop in people with MPS I and IVA. Over 4 years, 240 participants will get yearly heart ultrasounds, blood tests, and urine tests. The goal is to find reliable markers that can track heart health and guide future treatmen…
Matched conditions: MPS IVA
Sponsor: Children's Hospital of Orange County • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:14 UTC