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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

15.03.19

CTD Holdings Announces Plan to Launch Clinical Trial of Trappsol® Cyclo™ in Alzheimer’s Disease

ALACHUA, FL – (Globe Newswire) – March 14, 2019 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, announced today that the company has initiated planning for a Phase I clinical trial for treatment of Alzheimer’s Disease. The trial will use Trappsol® Cyclo™, the company’s proprietary formulation of hydroxypropyl beta cyclodextrin, administered intravenously.

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11.03.19

IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of Niemann-Pick Disease type C (NPC)

IntraBio Clinical Trial Authorization Application Approved by the MHRA for the Treatment of Niemann-Pick Disease type C (NPC): OXFORD, UK, March 11, 2019-- IntraBio Inc., a late-stage biopharmaceutical company, today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved its Clinical Trial Authorisation (CTA) Application for Clinical Trial IB1001-201 with its lead compound (IB1001) for the treatment of Niemann-Pick disease Type C (NPC).

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