A Global Prospective Observational Registry of Patients With Pompe Disease
About This Trial
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
Who May Be Eligible (Plain English)
Original Eligibility Criteria
View original clinical language
Treatments Being Tested
Cipaglucosidase alfa
Enzyme Replacement Therapy (ERT) via intravenous infusion
Miglustat
Participants received ATB200 co-administered with AT2221 (Miglustat)
Alglucosidase alfa or Avalglucosidase alfa
Patients prescribed other commercially available ERT after local regulatory approval
Untreated
Patients who are not currently receiving any medical therapy for Pompe disease.