Work Package 1

Title of Work Package: Standardized patient registry and natural history study

Objectives: WP1 is devoted to building an internationally harmonized patient registry for NBIA patients. Up to this point, only small local databases involving NBIA have existed; this has created large research obstacles in rare diseases such as NBIA. Together with the joint biobank (WP2), the standardized collection of patient data will provide the opportunity to overcome fragmentation of local patient collections and enable formation of large cohorts to catalyze basic and clinical research in the field.




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