POMPE DISEASE INFANTILE-ONSET
Clinical trials for POMPE DISEASE INFANTILE-ONSET 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: POMPE DISEASE INFANTILE-ONSET
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 26, 2026 06:11 UTC
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New hope for infants with pompe disease: experimental drug now available
Disease control AVAILABLEThis program offers early access to an experimental drug (ATB200/AT2221) for children with infantile-onset Pompe disease who are not getting better with standard treatment. It is for kids up to 18 years old who cannot join other clinical trials. The goal is to provide the drug wh…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated May 18, 2026 11:59 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects health information from up to 250 people with lysosomal storage diseases, including pregnant women whose fetus has a diagnosis. Researchers will track symptoms, lab results, and development over time to better understand how these rare conditions progress. The…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 26, 2026 06:09 UTC