International Niemann–Pick Disease Alliance

International Niemann-Pick Disease Registry

What is the INPDR?

Niemann Pick Diseases (NPD) are rare, progressive conditions with many unanswered questions. More information is needed to better understand how they affect people and progress over time. The International Niemann Pick Disease Registry (INPDR) – a joint initiative between patient organisations and clinicians involved in the care of people with NPD – was established to better understand NPD to help guide researchers so that potential treatments can be developed as fast as possible.

There are two sections in the INPDR, together collecting clinical, genetic, diagnostic and outcome data: patient reported data (PRD), completed by the patient or their carer if appropriate; and clinician reported data (CRD), completed through the hospital or medical centre you attend. Your information can be held in either the CRD, the PRD, or in both sections on the INPDR, with an individual’s records linked by an anonymous common identifier.

You can confidentially record your health information in the patient-reported section of the INPDR. Only anonymous health information will be made accessible to qualified researchers who are granted permission by the Steering committee. You will then be provided with guidance on how to get your clinical dataset (CRD) updated in the INPDR. This varies by country.

We welcome all patients diagnosed with any type of NPD worldwide. Patients can participate in the registry regardless of whether or not they are involved in other clinical studies and trials.

If you have any questions at all, please do contact us.

Why should I sign up?

With rare diseases like these, every patient counts. By joining information on a global level, we can meet the following goals:

  • For you – track your health, and in the future, if you choose to do so, compare yourself to a group of anonymous people with NPD in the database.
  • For doctors – help physicians better manage their patients by understanding how NPD affects the body and quality of life over time.
  • For treatment – help researchers design trials more quickly and develop potential treatments
  • For others – improve the standard of care for all people with NPD worldwide.

The clinician reported section of the INPDR is already live, with patients from Italy, the UK, Spain, Ireland and the Czech Republic enrolled, and many more following.

We’re working really hard to get the patient reported section up and running. Soon, we’ll be asking you to JOIN US, and enrol in the international registry. Watch out for updates, and please come back soon!

Join the Registry

Calling all patients and carers! We've created a global registry of all NPD patients - if you're not enrolled yet, join us now...

Think Again. Think NP-C

Join our international campaign to improve diagnosis of NP-C!