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
Pompe Disease
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
A Global Prospective Observational Registry of Patients With Pompe Disease
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University of Arkansas Medical Science, Little Rock, Arkansas, United States, 72205
University of California Irvine, Irvine, California, United States, 92697
Wolfson Children's Hospital, Jacksonville, Florida, United States, 32207
Emory University, Atlanta, Georgia, United States, 30322
Indiana University, IU Health Physicians Neurology, Indianapolis, Indiana, United States, 46202
Washington University School of Medicine, Saint Louis, Missouri, United States, 63110
NYU Neurogenetics, NYU Langone Medical Center, New York, New York, United States, 10017
Duke University Medical Center, Durham, North Carolina, United States, 27710
University of Cincinnati Medical Center, Cincinnati, Ohio, United States, 45219
Cincinnati Children's Hospital, Cincinnati, Ohio, United States, 45229
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
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ALL
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Amicus Therapeutics,
2034-12-20