Thank you for donating!

You can donate using the following services.

Our Friends

Here at NPUK you will regularly find us working with other like-minded organisations. We have a track-record of successful collaborations with organisations that share our focus and commitment to improving the lives of those affected with Niemann-Pick disease and other rare diseases.

We work and connect with other organisations both nationally and internationally, enabling us to develop a stronger, more united voice, to share knowledge and to access specialist information. Through our collaborations we will be able to achieve common goals, make a greater impact and accomplish more on behalf of those we support.

Our Friends

Support us

Latest news

08.09.16

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.

Read more

08.09.16

CTD Holdings Initiates Recruitment for Phase I/II Clinical Study in Europe of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C

“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
View all news Subscribe