Pompe Registry – Global Data Collection and Sharing of the Variability, Progression and Natural History of Pompe Disease

Summary

The goal of the Pompe Disease Registry is to collect clinical data on people with Pompe disease around the world in an effort to help healthcare professionals involved in the treatment or diagnosis of Pompe disease better understand it and its management, as well as evaluate the long-term effectiveness and safety of available treatment options and support measures including enzyme replacement therapy (ERT) with Myozyme®.

The Pompe Registry program was started worldwide in 2004 for patients with Pompe disease to help health care providers better understand that disease. Since there is no known cure for Pompe Disease, it is expected that the Pompe Registry will continue for a number of years. People with Pompe disease can participate in the Registry, regardless of whether they are receiving any treatment. The Registry is sponsored and run by Sanofi Genzyme.

The main purpose of the Registry is to collect health information to better understand rare diseases. Pompe Disease Registry findings may provide valuable information about Pompe disease and its medical management to your (or your child’s) doctor and other healthcare professionals. Since the Pompe Registry is an observational program, no experimental procedures are involved. Therefore, there are no medical risks to participants.