What is the INPDR?
We are an independent organisation dedicated to improving understanding and outcomes in Niemann-Pick diseases. As part of this work, we develop and manage disease-specific registries—structured platforms that systematically collect and curate patient and clinical data.
Through these registries, we bring together real-world data from across countries, enabling a morecomplete and reliable picture of Niemann-Pick diseases, including how they present, progress, andimpact individuals over time.
The data collected through our registries supports a wide range of research and evidence-generation activities. By providing controlled access toanonymised, curated datasets, we help clinicians, researchers, patient organisations, industry partners, and other stakeholders generate insights thatadvance scientific knowledge, improve understanding of the patient experience, inform healthcare decision-making, and support the development ofnew treatments and interventions.
For ultra-rare conditions, this kind of coordinated data collection is critical. It helps address key evidencegaps, supports earlier and more accurate diagnosis, informs clinical care, and strengthens the evidencebase needed for research, treatment development, and decision-making.
The INPDR registries cover Acid Sphingomyelinase Deficiency (ASMD, types A & B) and Niemann-Pickdisease type C (NPC). By capturing data that reflects real patient experiences, we enable insights that canimprove understanding, guide care, and ultimately contribute to better outcomes for the community.
