POMPE DISEASE INFANTILE-ONSET
Clinical trials for POMPE DISEASE INFANTILE-ONSET explained in plain language.
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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 17, 2026 00:52 UTC
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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: POMPE DISEASE INFANTILE-ONSET
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated May 13, 2026 15:59 UTC
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New registry aims to unlock secrets of rare lysosomal diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 people with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to learn how these conditions develop and how they appear before birth. Researchers will review medical records and test samples t…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated May 17, 2026 00:47 UTC