POM-005
Brief description of study
A GLOBAL PROSPECTIVE OBSERVATIONAL REGISTRY OF PATIENTS WITH POMPE DISEASE
Detailed description of study
Objectives:
•To evaluate the long-term safety of Pompe disease treatments through collection ofdata that describe the frequency of AEs/SAEs occurring in Pompe disease patients,including IARs, hypersensitivity reactions (including anaphylaxis), immune complexrelated reactions, medication errors in the home infusion setting, and pregnancyexposures
•To evaluate the long-term real-world effectiveness of Pompe disease treatments
•To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and PROs
•To describe the natural history of untreated Pompe Disease
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Medical Research
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Age: - 99 Years
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Gender: All
Inclusion Criteria
•Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzymeactivity and/or GAA genotyping
•Appropriate informed consent/assent obtained for participation in the registry
Exclusion Criteria
•Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an EAP has context menu
