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Recruiting
NCT07834320
Pompe Disease RWE Long-term Monitoring Program
Conditions: Pompe Disease
Sex: All
Healthy volunteers: No
Enrollment: 40
Sponsor: National Taiwan University Hospital
Location: National Taiwan University Hospital Taipei Taiwan
Summary
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.
Eligibility Criteria
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 \
Source: ClinicalTrials.gov (NCT07834320). StuddyBuddy aggregates publicly available trial information.
