Approximately 350 million people are affected by a rare disease – that’s an estimate of almost 8% of the global population. Despite these startling figures support for those affected by rare diseases is often found lacking due to combination of an absence of awareness, clinical knowledge, and funding.
Due to its rarity Niemann-Pick disease, like other rare conditions, poses unique challenges. The small number of affected patients results in limited clinical experience. However it is our hope that the International Niemann-Pick Disease Registry (INPDR) will change this for the better.
Participating in the INPDR, by sharing your data, is one of the most important things you can do to influence progress in research and care for Niemann-Pick disease patients now and in the future. Patients taking part around the world will provide robust and useful data on this rare disease, help to develop and recruit to new clinical studies and support access to new and emerging therapies.
As well as sharing your clinical data, you can also share your experience of living with Niemann-Pick Disease and the wider impact on your quality of life by adding Patient Reported Data. Your perspective is incredibly important in influencing progress in care, treatment and research.
Taking part in the INPDR requires no extra testing or lifestyle changes. With your consent, your clinical team will be in charge of inputting into the registry and maintaining the Clinician Reported Data. You can add to and update your Patient Reported Data whenever it suits you. You are not obliged to take part in the registry, and your quality of care will not be at all affected if you do not take part. Participating in the registry is 100% voluntary.