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← Trials
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Pompe Disease RWE Long-term Monitoring Program

Recruitingn/aAges any - anyAllNational Taiwan University Hospital
NCT07834320 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

Evaluate short- and long-term biomarker, motor function, respiratory function and safety with avalglucosidase alfa in IOPD and LOPD patients who switched from other ERT.

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📋 Eligibility rules (as published)
Inclusion criteria: -Confirmed GAA enzyme deficiency (any tissue source) and/or confirmed 2 pathogenic GAA gene variants Pompe patient Exclusion criteria: * Having severe allergic reaction to avalglucosidase alfa. * Patient/parent/legal guardian is not agreeing to provide signed informed consent, and the patient, if \<18 years of age
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What it tests

  • Not listed
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Where

1 site
National Taiwan University Hospital
Taipei, Taiwan
🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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