About the Registry

The INPDR is a patient registry that collects clinical and patient reported data in a registration system to support the needs of researchers and clinicians, towards the ultimate aim of improving the lives of those affected by Niemann-Pick diseases.

The INPDR is a truly global collaborative environment underpinned by robust governance.

The Registry is made up of Clinician Reported Data (CRD) and Patient Reported Data (PRD). Both are collected electronically through a secure, web-based platform, see more information on the differences between the PRD and the CRD here.

The collection of extensive clinical and patient-reported data is actively supported by clinicians, patient advocates, and researchers from 18 countries, across 5 continents, facilitating the collection of demographic information, diagnostic results, treatment, and health outcomes data.

Patient Reported data is collected through a questionnaire that asks patients and/or carers about patient experience to capture wider disease impact for patients and their families, the questions are tailored to the patient responding, whether an individual or carer, ASMD or NPC, adult or child.