Thank you for donating!

You can donate using the following services.

Get Involved

Without people like you getting involved, we wouldn't be able to provide the care and support that we provide to families affected by Niemann-Pick disease. There are numerous opportunities available when it comes to helping us, everything from taking part in our fun days as a member of our passionate volunteer team, setting up fundraisers, or drumming up interest and donations through creative sponsored events. By working together we can make a real difference to the lives of those people affected by Niemann-Pick disease. Contact us if you would like to know more.

Get Involved

Support us

Latest news

25.08.16

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

25.08.16

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