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First received: November 02, 2023

Clinical Trial: A Global Prospective Observational Registry of Patients With Pompe Disease

Study Status: NOT_YET_RECRUITING

Recruit Status: NOT_YET_RECRUITING

Condition: Pompe Disease

Study Type: OBSERVATIONAL


Official Title: A Global Prospective Observational Registry of Patients With Pompe Disease

Brief 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…

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