The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with 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 with Myozyme®.
(Text source: www.clinicaltrials.gov)