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Reporting. Collaborating. Advancing.

Helping to improve the quality of care for patients with the rare diseases Gaucher, Fabry, MPS I and Pompe with over 650 investigators and 13,500 patients and counting!

RegistryNXT! is a novel, web-based data collection and reporting technology platform that underlies the Sanofi Genzyme Rare Disease Registries. The Rare Disease Registries are a global, observational, and voluntary program designed to collect patient clinical data related to the onset, progression, and treatment course of Gaucher, Fabry, MPS I and Pompe diseases. Publication of these data in peer-reviewed biomedical journals is a major emphasis of the Rare Disease Registries. In addition, such data are used to fulfill various global regulatory commitments of Sanofi Genzyme. Regional and International Advisory Boards provide scientific oversight and direction to the Rare Disease Registries. Advisory Board members are physicians with expertise in the diseases included in the Rare Disease Registries and serve as liaisons between the Rare Disease Registries and the respective medical communities.

Researching Lysosomal Storage Disorders

A disease registry can help improve disease understanding by enabling physicians to:

  • Contribute and share data by entering participating patient data and having the ability to report on and share that data with other practitioners or their patient in near real time.
  • Gain insight and understanding of the variations in the disease across multiple patients and assessing an individual patient’s progress compared to that of other patient’s, through aggregate data reporting.
  • Improve research globally by contributing their data to aggregate analyses to help understand progression of the disease and ultimately improve patient care.
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