When a disease is rare, every patient’s experience matters.
The International Niemann-Pick Disease Registry (INPDR) brings together information from people living with Acid Sphingomyelinase Deficiency (ASMD) and Niemann-Pick disease type C (NPC) around the world.
By participating, patients and families help build a clearer understanding of Niemann-Pick disease — how it affects people, how it changes over time, and how treatments and care impact patients’ lives.
Because ASMD and NPC are rare, every contribution helps strengthen what researchers and clinicians can learn about these diseases.
The more we learn from patients and families, the stronger the foundation for future research and better care.
INPDR data and collaborations have contributed to peer-reviewed publications, scientific presentations, conference abstracts and posters, clinical resources, and other research focused on ASMD and NPC.
This work is helping researchers better understand Niemann-Pick disease, patient and caregiver experiences, disease progression, treatment outcomes, and clinical care.
See how registry data is being put to work.
There are two ways information is contributed to the INPDR:
Patients, parents, caregivers, or guardians can participate directly online by sharing information about quality of life and the experience of living with Niemann-Pick disease.
Create an account, provide consent, complete your first questionnaire, and periodically update your information over time.
With your consent, your healthcare team can contribute clinical information such as diagnosis, symptoms, treatments, and disease progression.
Patient-reported and clinician-reported information together can provide researchers with a more complete picture of Niemann-Pick disease.
For rare diseases like ASMD and NPC, building meaningful knowledge depends on participation from patients and families around the world.
Your experience adds to a growing body of knowledge that helps researchers better understand Niemann-Pick disease and advance research for the future. It takes an entire community, across geographic boundaries, diagnoses, ages, and experiences — working together to build the knowledge needed to move research forward.
The safety and security of your data is our number one priority.
The data held on the Registry is anonymous and will not contain any personal identifiable information such as names or addresses.
The clinical team who look after you will be able to identify your data by linking this to a unique subject number which will be given when you enroll onto the Registry. Access to the registry will be restricted to approved individuals who have made an application to be a part of the registry, and approval will only be given after the application is reviewed by the INPDR scientific advisory board.
The INPDR is a global registry and a not-for-profit, registered company, based in the UK.
The initiative encourages global collaboration between patient groups, clinicians, scientists and researchers, all of whom wish to improve care and treatment options for NPD patients everywhere.
Please visit the website inpdr.org or follow us on Facebook, Twitter or LinkedIn.
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