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.Read more
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.Read more
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.Read more
“We are pleased to be participating in this promising clinical trial,” said Dr. Sharma. “Based on the compelling compassionate use data generated to date, we believe that Trappsol® Cyclo™ administered intravenously has the potential to effectively treat NPC, for which a significant unmet need exists, with only one approved treatment in Europe and none in the U.S. We look forward to evaluating Trappsol® Cyclo™ in the clinical setting in order confirm these encouraging results.”Read more