The INPDR - your registry

The Patient Reported Database

The Patient Reported Database (PRD) is part of the International Niemann-Pick Disease Registry (INPDR). It brings together information shared directly by patients and families to help build a clearer understanding of what it is really like to live with Niemann-Pick diseases.

While clinical data helps describe the medical aspects of the condition, the PRD captures something equally important—your lived experience. This includes how symptoms affect daily life, how the disease changes over time, and the impact it has on individuals and families.

For rare diseases like Niemann-Pick, this kind of insight is essential. By contributing your experience, you help ensure that research and care are shaped not only by clinical data, but by the realities of day-to-day life. This supports better understanding, more relevant research, and ultimately improvements in care and treatment.

How to join the Patient Reported Database

Taking part in the PRD is straightforward and designed to be accessible and secure.

1. Create your account

Start by setting up a secure online account via the INPDR sign-up page. This allows you to safely access and manage your information.

2. Give informed consent

You will be provided with a clear information sheet and consent form. This explains how your data will be used, stored, and protected, so you can make an informed decision about taking part.

3. Verification

To ensure the accuracy and quality of the registry, the INPDR team will verify your diagnosis status. This step helps make sure that all data is used appropriately and meaningfully.

4. Complete your first questionnaire

Once registered, you will be invited to complete your first questionnaire. This is where you begin sharing your experience of living with Niemann-Pick disease, in your own time and at your own pace.

5. Ongoing updates

You will be asked to return approximately every six months to update your information. These updates are important for understanding how the condition changes over time.

Linking with the Clinical Research Database

To create a more complete picture, your information in the PRD can be complemented by clinical data from your healthcare provider through the Clinician Reported Database (CRD).

You can speak to your clinician about enrolling in the CRD. If they are not already participating, they can find out how to join and contribute. Combining patient-reported and clinician-reported data helps strengthen understanding of the diseases and increases the impact of every contribution.

Patients & families