International Niemann–Pick Disease Alliance
Update from the International Niemann-Pick Disease Alliance (INPDA):
The INPDA has been working hard throughout the year to facilitate progress for those affected by Niemann-Pick disease, by engaging in three key projects: the International Niemann-Pick Disease Registry (INPDR), the Think Again. Think NP-C Campaign and the Loire Valley Meeting (LVM). Each aim to make a difference in the areas of scientific, clinical and therapeutic research, patient care, access to treatments and time to diagnosis.
The International Niemann-Pick Disease Registry (INPDR) Project has reached its final year and enjoyed its official launch in June 2016, at the Institute of Translational Medicine, Queen Elizabeth hospital, Birmingham. Six countries were represented at the launch, which provided the opportunity for the project partners to share outcomes, achievements and learning points with project stakeholders, including patients and carers, patient associations, health policy makers, health care professionals, industry and researchers. In addition, Mum Stefanie Rymsza, whose daughter Danielle was the first patient to be enrolled in the INPDR in the UK, gave a presentation explaining how important the INPDR is to patients and families.
The INPDR is now live, with six countries enrolling a total of 250 patients so far. With the introduction of Patient Reported Data entry, patients now have the power to enrol in the INPDR themselves. To learn more about how you can make a difference by joining the INPDR visit www.inpdr.org or contact your national patient group – in the UK, contact NPUK by email info@npuk.org or telephone 0191 415 0693.
Earlier this year, the Think Again. Think NP-C (TATN) Campaign stand was present at the European Academy of Neurology (EAN) conference in Copenhagen on 27 – 29 May 2016 and also at the Study of Inborn Errors of Metabolism (SSIEM) conference in Rome from 5 – 8 September 2016, where the Campaign celebrated its second birthday!
The TATN Campaign has now been successfully launched in 16 countries, with the campaign messages being heard by relevant healthcare professionals around the world.
The INPDA is grateful to everyone involved in supporting the continued progression of this valuable campaign, which aims to raise awareness of the symptoms of NP-C and reduce time to diagnosis.