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Clinical Trial Summary

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: - To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients - 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 patient-reported outcomes (PROs) - To describe the natural history of untreated Pompe disease


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT06121011
Study type Observational [Patient Registry]
Source Amicus Therapeutics
Contact For Site
Phone 215-921-7600
Email PompeSiteInfo@amicusrx.com
Status Recruiting
Phase
Start date February 16, 2024
Completion date December 20, 2034

See also
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