As a small charity, we rely on grants and voluntary donations to ensure that we can continue helping the individuals and families in our community. As a result we are forever grateful to our amazing grant providers, who enable us to continue our work supporting those affected by Niemann-Pick disease.
Our grant providers include:
BBC Children in Need:
BBC Children in Need is very important to us here at NPUK; their generous grant funding programme has provided part-funding for our Clinical Nurse Specialist since 1999. This has enabled us to continue providing much-needed support to children and families affected by Niemann-Pick diseases – support that is not available elsewhere and would not exist without the kindness and generosity of all those donating to the BBC Children in Need Appeal each year.
Pictured: Shona Beveridge (17, NP-C), with The One Show’s Matt Baker and Alex Jones – Shona took part in the Rickshaw Challenge 2017 in support of BBC Children in Need.
The Big Lottery Fund:
We are now approaching the end of the second year of our Big Lottery funded project, “Shaping our Future Together”. The aim of this five year project is to improve and implement new support services and resources that will help us more effectively reach those affected by Niemann-Pick and their families.
Pictured: NPUK Project Team Leader Louise Metcalfe (second on the right) at our Annual Family Conference. Louise’s post, as well as NPUK: Project Families Officer Steve Neal, are fully funded by The Big Lottery Fund.
The Hollie Foundation:
NPUK is deeply grateful to The Hollie Foundation for its continued support of our work through grant funding in 2017/2018. The Foundation has generously awarded further grant funding to part support the post of NPUK’s Senior Families Advocate, held by the very dedicated Elizabeth Davenport. This is the fourth consecutive year that The Hollie Foundation has supported this essential post, enabling Elizabeth to provide a high-level of individualised non-clinical support to those affected by all types of Niemann-Pick disease and their families. In addition, The Hollie Foundation has also kindly granted much-needed funding to cover the cost of the Children and Young Persons’ Activity Programme and our Annual Family Conference and our regional family get-togethers.
Pictured: The Hollie Foundation Co-founder and NPUK Trustee Helen Carter (third from the left) represented NPUK at the 5th Biennial “Face to Face” International Niemann-Pick Disease Alliance (INPDA) Meeting 2017.
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