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What We Do

How We Help

We make a positive difference to the lives of those affected by Niemann-Pick disease, through:

  • Providing support and information regarding all aspects of Niemann-Pick disease, via a 24-hour help line, educational literature, our website, regular newsletters and e-bulletins.
  • Our Clinical Nurse Specialist who provides expert care and practical advice, plus home visits whenever necessary. Genetic counselling and advocacy services are also provided.
  • Our Families Officer who provides non-clinical advice and information, offering an individual advocacy service which includes home visits and assistance at clinic appointments.
  • Promoting relevant research, raising awareness and offering education and information to carers, associated professionals and the general public.
  • Our Annual Family Conference, comprising of adult and children’s programmes. The Conference highlights the most recent advances in research, and provides an ideal opportunity to strengthen our family support network.

Watch the video below to see first-hand accounts of our impact on our community, and find out more by visiting our ‘Care and Support’ pages, here.

Latest news

30.01.19

Orphazyme reports positive results from full data set of Phase II/III arimoclomol trial in Niemann-Pick disease Type C (NPC)

We are pleased to share the latest update from Orphazyme entitled 'Orphazyme reports positive results from full data set of Phase II/III arimoclomol trial in Niemann-Pick disease Type C (NPC)'.

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11.01.19

CTD Announces Presentation at Upcoming Conference on Lysosomal Storage Diseases

CTD Holdings: Annual conference offers opportunities for academic researchers and industry representatives to exchange information on novel therapeutic interventions...

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