Pompe Disease Registry

The Pompe Registry is an ongoing, international multi-center, strictly observational program
that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of
treatment status. No experimental intervention is involved; patients in the Registry undergo
clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

- To enhance the understanding of the variability, progression, and natural history of the
key manifestations of Pompe disease;

- To assist the Pompe medical community with the development of recommendations for
monitoring patients and reports on patient outcomes to help optimize patient care;

- To characterize and describe the Pompe disease population as a whole; and

- To evaluate the long-term effectiveness and safety of available treatment options
including ERT(Enzyme Replacement Therapy) with Myozyme®.