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Care and Support

Our main focus, above all else, is to provide unique care and support services to families affected by Niemann-Pick diseases. We originally started out as a parent support group made up of ten families affected by Niemann-Pick disease and 25 years later we stand stronger that ever.

Since 1991 we have continually developed our service so we can better serve those who need it most. Many of the individuals who work within the charity have been directly affected by Niemann-Pick in their personal lives, which adds a unity and shared-understanding of the potential struggles and subsequent help you may need.

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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.

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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.”

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