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What is the INPDR?

We are a disease-specific registry created by our community for our community – benefiting patients and families but also clinicians, researchers and other key stakeholders.

Having an international registry for Niemann-Pick diseases is particularly important given their ultra-rare status. There’s a real need for disease-specific information to support diagnosis, improve clinical care and advance research.

The INPDR encompasses Acid Sphingomyelinase Deficiency (ASMD, types A & B) and Niemann-Pick disease type C (NPC). We record information that accurately reflects the patient experience – in order to improve that experience.

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