Our History

The rare disease environment of today looks entirely different from 2013 when the INPDR was launched. Over the years, high-quality disease-specific data has become increasingly important to reflect the patient experience, support stakeholders in addressing unmet needs, and improving patient outcomes. With this in mind, since 2013, the INPDR has continued to evolve, ensuring that we keep pushing for improvements in the care of Niemann-Pick diseases internationally.

In 2013 the International Niemann-Pick Disease Registry (INPDR) was launched with grant funding from the Consumers, Health, Agriculture and Food Executive Agency (CHAFEA- an executive of the European Union).

The resulting three-year grant project was supported by eleven professional partners (seven Clinical centres and four patient organisations) in seven EU countries, alongside seventeen associate partners from the rest of the world.

This project successfully resulted in a multifunctional web-based registry capturing clinical data and contributing to effective clinical management, providing a platform for future research launched in 2014.

The EU project was successfully completed in 2016 and, recognising the need to sustain this invaluable resource, the International Niemann-Pick Disease Alliance (INPDA) supported the INPDR in achieving independent status as a non-profit charitable company in 2017, constituted under UK law.

The INPDR is now established as an independently governed rare disease registry for the global collection of Niemann-Pick patient data, encompassing both Acid Sphingomyelinase Deficiency (ASMD type A and B) and Niemann-Pick disease type C (NPC).