International Niemann–Pick Disease Alliance
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.
With rare diseases like these, every patient counts. By joining information on a global level, we can meet the following goals:
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!