Thank you for donating!

You can donate using the following services.

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

11.06.19

Orphazyme to prepare for filing of arimoclomol in Europe for Niemann-Pick disease Type C (NPC)

Orphazyme plans to meet with the US Food and Drug Administration (FDA) to discuss the pathway forward during the Summer and will provide an update after it has received written comments from the FDA...

Read more

03.06.19

CTD Holdings Closes $7.4 Million Private Placement

CTD Holdings, Inc. (OTCQB: CTDH)("CTD" or the "Company"), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease with unmet medical need, today announced that it closed its previously announced private placement of securities with a group of institutional and accredited investors that included directors and officers of the Company.

Read more
View all news Subscribe