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A Global Prospective Observational Registry

Neurology, Genetics
Tahseen Mozaffar
A Global Prospective Observational Registry of Patients with Pompe Disease
Brain - Neurologic/ Psychologic
Neuromuscular Diseases
Pompe Disease

Study Description

Eligibility

  1. Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
  1. Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
A female nurse attentively holds a tablet, ready to assist with patient care and medical information.

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