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  • NPDR: An EU Rare Diseases Registry for Niemann-Pick Disease Type A, B AND C (NPDR) Rochester, Minn.

    The purposes of this study are to establish the natural history of the 3 diseases including their characteristics, management and outcomes; to assess clinical effectiveness of management and quality of care; to provide an inventory of patients for recruitment to interventional studies; to establish genotype-phenotype correlations; and to support post marketing obligations for approved therapies.

Closed for Enrollment