Groundbreaking trial aims to treat fatal diseases in the womb
NCT ID NCT04532047
First seen Mar 26, 2026 · Last updated May 12, 2026 · Updated 6 times
Summary
This 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 is to see if this early treatment can reduce disease effects, but children will still need lifelong management after birth.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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University of California
RECRUITINGSan Francisco, California, 94158, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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