What is the INPDR?

The International Niemann-Pick Disease Registry (INPDR) serves as a crucial platform for driving research, supporting better clinical care, and connecting the global Niemann-Pick community. Through the collection and study of worldwide data, the INPDR helps to deepen our knowledge of Acid Sphingomyelinase Deficiency (ASMD Types A & B) and Niemann-Pick Type C (NPC). Unlike traditional rare disease registries, the INPDR brings together patient information in one international resource, offering unique insights into these conditions.

Now operating as an independently governed global registry, the INPDR captures and manages essential data on both ASMD and NPC, helping researchers, clinicians, and families work towards improved understanding, treatment, and care.

What is the Purpose of the INPDR?

The International Niemann-Pick Disease Registry (INPDR) exists to create and maintain a unique, patient-led global database that drives research, supports the development of therapies, and enhances care for people living with Niemann-Pick diseases (ASMD and NPC). By fostering worldwide collaboration, the registry provides a powerful tool to accelerate understanding and improve outcomes for patients.

The data collected within the INPDR contributes to:

  • Enabling clinical research through data sharing and analytical support
  • Mapping the epidemiology and natural history of ASMD (Types A & B) and NPC
  • Identifying genotype-phenotype correlations
  • Speeding up recruitment for interventional studies
  • Advancing scientific knowledge and encouraging the education of healthcare professionals and researchers
  • Supporting the creation of diagnostic standards, clinical management strategies, and improved quality of care
  • Providing evidence for advocacy, public awareness, and policy-making
  • Strengthening collaboration between patients, clinicians, and researchers worldwide