POMPE DISEASE INFANTILE-ONSET
Clinical trials for POMPE DISEASE INFANTILE-ONSET explained in plain language.
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New hope for babies with rare muscle disease when standard treatment fails
Disease control AVAILABLEThis program provides access to an experimental drug combination (ATB200/AT2221) for children with infantile-onset Pompe disease who are getting worse on their current enzyme replacement therapy. The treatment aims to better control this rare genetic disorder that affects muscles…
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Sponsor: Amicus Therapeutics • Aim: Disease control
Last updated Apr 01, 2026 14:41 UTC
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Doctors test Life-Saving enzyme treatment for babies before they are born
Disease control Recruiting nowThis early-stage study is testing whether giving enzyme replacement therapy to fetuses still in the womb is safe and feasible. It aims to treat rare, severe genetic disorders that often cause life-threatening complications before or shortly after birth. The hope is that treating …
Matched conditions: POMPE DISEASE INFANTILE-ONSET
Phase: PHASE1 • Sponsor: University of California, San Francisco • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC