The following family stories, developed by Packer Forbes on behalf of Actelion Pharmaceuticals, aim to raise awareness of Niemann-Pick Disease Type C (NP-C) amongst healthcare professionals and the public, to improve recognition of symptoms and to reduce feelings of isolation in those affected.
These personal stories highlight the impact that NP-C has on Hollie and Annie, as well as their families.
Annie’s Story – Living with a Genetic Condition
Hollie’s Story – Our Journey to Diagnosis
Summary of Hollie’s and Annie’s Stories
Summary of the work of NPUK
In 2011, NPUK was delighted to have been awarded £25,000 in recognition of its work to support people affected by Niemann-Pick diseases. The Charity beat more than 400 organisations from around the UK to become one of ten winners of the GlaxoSmithKline (GSK) IMPACT Awards, run in partnership with health think-tank The King’s Fund. The following short films gives an overview of the work undertaken by NPUK.
14.10.19
Study Title: A Phase 2b/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects with Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease...
Read more13.09.19
ALACHUA, FL–(Businesswire)–13 September 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, today announced that the company will provide Trappsol® Cyclo™, its proprietary hydroxypropyl beta cyclodextrin drug, to a pediatric patient diagnosed with Niemann-Pick Disease Type C...
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