NPUK encourages and promotes scientific and medical research into Niemann-Pick disease. We want to make it easier for research into Niemann-Pick disease to take place, and where appropriate, to enable UK patient participation in clinical trials. Due to the expense, expertise and other resources required to conduct research and clinical trials, we focus our activity on enabling and supporting these activities, rather than conducting them ourselves.
The NPUK Board of Trustees will consider applications for research funding as listed below, and will determine the nature of research to be undertaken in consultation with its Research Co-ordinator and Medical Adviser. Where appropriate, guidance will be sought from an external expert scientific advisory board:
This annual award of up to £1,000 is made available to an individual who is engaged in either research, teaching, treatment or care in the field of Niemann-Pick disease, within the public or private sectors in the UK. The award is granted in response to the submission of a research project (not a literature review), which provides an original contribution to the scientific or public understanding of Niemann-Pick disease and/or its treatment.
How to apply
We are now inviting applicants to submit for the Peter Carlton Jones Memorial Award 2019…so if you have been taking part in a Niemann-Pick disease-based research project then find out how to apply below.
Submissions should be in the form of an abstract outline, on one side of A4 paper (approximately 600 words), which includes clear statements on the work undertaken, methodology, findings, and any principal features in regard to the nature of the project. Submissions should be made to:
Suite 2, Vermont House
Concord, Washington NE37 2SQ
Tel: 0191 415 0693
Closing date for submissions: 31st July 2019
Applications will then be shortlisted by the Awards Committee, which may then lead to the potential requirement of further information and/or a project report, prior to reaching a final decision.
The successful applicant will be invited along to the NPUK Annual Family Conference and Interactive Workshop to receive their award and also to meet patients and their families and healthcare professionals associated with Niemann-Pick disease.
*Peter Carlton Jones was a highly distinguished and respected scientist. He was a leading figure in mentoring research students, and it is in honour of his name that this prize is awarded.
Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”Read more