International Niemann Pick Disease Registry

The International Niemann-Pick Disease Registry (INPDR) is a not-for-profit charitable company based in the UK, with an independent Board of Trustees and Scientific Advisory Committee. This single disease-specific registry was created by a collaboration of patient groups, health and research professionals with the aim of improving standards of diagnosis, care, and treatment for patients affected by Niemann-Pick diseases on a global basis.

The aims and objectives of the INPDR are:

• To establish the natural history of ASMD Niemann-Pick disease and Niemann-Pick type C 
• To establish genotype-phenotype correlations
• To assess standards of diagnosis, clinical management and quality of care
• To support recruitment to interventional studies
• To encourage education of health professionals
• To empower patients
• Support regulatory interaction between national/supranational medicines agencies and pharmaceutical companies
• Speed up progress and understanding of NPD