Thank you for donating!

You can donate using the following services.

Research

Alongside providing support and care for those living with or affected by Niemann-Pick disease, NPUK also provides information relating to research. NPUK shares knowledge, expertise and works in partnership and collaboration with other not-for-profit organisations, medical authorities and scientific institutions across the world, in order to facilitate progress towards therapeutic interventions for the Niemann-Pick disease.

Where possible, NPUK also funds appropriate research in order to improve medical and scientific knowledge and promote understanding of the disease. The results are often shared amongst our community of families at the annual family conference we hold every September. It is through this medical research and clinical trials that we will find innovative new ways in which to better manage Niemann-Pick disease.

Research

Support us

Latest news

25.08.16

Update #17 on the AIDNPC Clinical Programme

The study was recently completed in May 2017. A total of 35 patients were enrolled across 12 sites in 8 countries. The primary objective of the 001 study was to characterize the individual patient’s disease progression profile through clinical, biological and quality-of-life measures recorded prospectively, as well as the historic disease information collected from patient medical records. The 001 study has already provided us with some valuable learnings: The NPC-CSS disease score were completed by patients with a good compliance, the skin biopsies and blood tests have been valuable for development and conformation of biomarkers, and we found that two-thirds of patients were treated with miglustat as part of their standard of care. Once all data have been analysed, we expect to gain insight about biomarkers and how the disease evolves over time in greater detail.

Read more

25.08.16

Route of Administration Preferences Study

There is an opportunity for you to participate in a market research study about Niemann-Pick Type C, conducted by an organization called ApotheCom on behalf of an industry sponsor.

The goal of the study is to understand your preferences and concerns regarding the method through which a drug could be administered to manage Niemann-Pick Type C.

Read more
View all news Subscribe