A Global Prospective Observational Registry of Patients With Pompe Disease

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

Eligibility

Age range

Not specified

Sex

All

Healthy volunteers

No

Interventions

• Biological
  Cipaglucosidase alfa

  Enzyme Replacement Therapy (ERT) via intravenous infusion

• Drug
  Miglustat

  Participants received ATB200 co-administered with AT2221 (Miglustat)

• Biological
  Alglucosidase alfa or Avalglucosidase alfa

  Patients prescribed other commercially available ERT after local regulatory approval

• Other
  Untreated

  Patients who are not currently receiving any medical therapy for Pompe disease.

Locations (41)