News

MRC Festival of Medical Research - Patient Open Day

On Friday 23rd June, as part of the annual MRC Festival of Medical Research, the MRC Harwell Institute will host a Patient Open Day. Patients, their families and representatives who are interested in finding out more about primary scientific research, the relationship between genes and disease, and how mice are used in medical research are invited to attend.

PLEASE HELP: Feedback for NHS England

If you or your family member(s) receive care and services linked to an ultra-rare disease, such as Niemann-Pick, we need your help.

In order to bring about change and improvement in the care and services you receive, Niemann-Pick UK has been asked to provide feedback to NHS England. By participating in a short, but highly important survey, you could help to influence this process and enhance future care for those affected by Niemann-Pick and other ultra-rare diseases.

NP-C Quality of Life Questionnaire – Can you Help?

You may recall that Niemann-Pick UK has been supporting Aston University in their work to deliver a research study titled “Development of health-related quality of life scales for patients and parents affected by Niemann-Pick Disease Type C”, which aims to bring benefits for the NP-C community.

This study has ethical and R&D approvals through the appropriate UK academic channels and in order to make further progress, research student Lydia needs your help.

An Exciting Announcement from Vtesse

Vtesse have announced an agreement to be acquired by Sucampo Pharmaceuticals—a global biopharmaceutical company that, like Vtesse, is focused on pursuing scientific innovation with a passion for improving the lives of patients.

WE NEED YOUR HELP: Westminster Hall Debate

WE NEED YOUR HELP: Just last week, NICE announced its decision to implement an upper funding threshold for its Highly Specialised Technology Programme, the main route used to evaluate medicines for rare disease patients. If implemented, the plans will significantly affect rare disease patients and their ability to access life-changing treatment, delivered at a time when we should be expanding access routes rather than further limiting them.