A Global Prospective Observational Registry of Patients With Pompe Disease
A Global Prospective Observational Registry of Patients With 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:
Inclusion Criteria:
Exclusion Criteria:
patientadvocacy@amicusrx.com609-662-2000
patientadvocacy@amicusrx.com609-662-2000
Indianapolis, Indiana 46202, United States
Philadelphia, Pennsylvania 19104, United States
Fairfax, Virginia 22030, United States
Bari, 70124, Italy
Naples, 80131, Italy
Torino, 10126, Italy
Udine, 33100, Italy
Rotterdam, 3015 GD, Netherlands
Ljubljana, 1000, Slovenia