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Pompe Registry - A Decade of Making a Difference

Posted on: April 11, 2016

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

Youtube video by Genzyme Corp., published April 11, 2016
Link: https://www.youtube.com/watch?v=COwyrHlDhXg

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