POMPE DISEASE INFANTILE-ONSET
Clinical trials for POMPE DISEASE INFANTILE-ONSET explained in plain language.
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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: POMPE DISEASE INFANTILE-ONSET
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Jun 27, 2026 11:02 UTC
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New hope for kids with pompe disease: expanded access to experimental therapy
Disease control AVAILABLEThis expanded access program offers a new treatment combination (ATB200 and AT2221) to children with infantile-onset Pompe disease who are not eligible for other clinical trials and are declining on standard enzyme replacement therapy. The goal is to provide access to this experi…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Jun 26, 2026 16:13 UTC
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New registry aims to unlock secrets of rare childhood diseases
Knowledge-focused Recruiting nowThis study collects information from up to 250 patients with lysosomal storage diseases (like certain forms of MPS, Pompe, Gaucher, and Wolman disease) to understand how these conditions develop and respond to treatments given before birth. Researchers will track symptoms, lab re…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: University of California, San Francisco • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:05 UTC