Pompe Registry – A Decade of Making a Difference

In 2014, the Pompe Registry recognized and celebrated an important milestone: the 10-year anniversary of the Registry. Started in 2004 and sponsored by Genzyme, a Sanofi company, the Pompe Registry was established as a global, voluntary, observational database to collect and report on the natural history and outcomes of patients with Pompe disease through routine clinical practice, regardless of patient treatment status. This video highlights the importance of the Registry through interviews with physicians in the field and patients with Pompe disease. (Source: https://www.youtube.com/watch?v=COwyrHlDhXg)

The video includes statements of Pompe medical professionals and Pompe patients participating in the registry:

Dr. Mark Roberts, UK
Dr. Sharon Krueger, First Pompe Registry Patient
Dr. Priya Kishnani, USA
Dr. Mary-Alice Abbott, USA
Dr. Edward Cupler, Saudi Arabia
Dr. David Stockton, USA
Prof. Antonio Toscano, Italy
Prof. Juan Llerena, Brazil
Abby Phelps, Pompe Registry Patient
Prof. Ans van der Ploeg, The Netherlands
Dr. Barry Byrne, USA
Dr. Yin-Hsiu Chien, Taiwan
Dr. Kenneth Berger, USA
Prof. Wolfgang Müller-Felber, Germany
Dr. Paula Clemens, USA
Dr. Virginia Kimonis, USA
Dr. Laura Case, USA

The Pompe Registry is an important program that Genzyme sponsors and administers. A disease registry contains medical information on patients with a specific condition, in a database that can be analyzed and used by physicians treating patients with the same condition. Registries have proven to be especially valuable in rare diseases like Pompe disease. Information submitted to the Pompe Registry will be maintained as confidential in accordance with applicable national privacy regulations and other state and local laws related to medical information. (Source: https://pompe.com/patients/pompe-registry.aspx)

More information

Information for patients on the Registry website: https://www.registrynxt.com/Home/For-Patients-and-Families
Information about the objectives and other aspects of the Registry (for Healthcare Professionals): https://www.pompe.com/healthcare-professionals/pompe-registry.aspx
Registration for Healthcare Professionals on the Registry website: https://www.registrynxt.com/Public-Registration