03.01.18
NPUK Chair, Will Evans, gives his 2018 message...
Read more13.12.17
CTD Holdings, Inc. today announced that it has received rare pediatric disease designation from the FDA for its Trappsol® Cyclo™ drug...
Read more20.11.17
A letter to the NPC community from Orphazyme’s CEO, Anders Hinsby. We are please to also provide translations (from English into German, French, Spanish, Italian, Polish) of this letter.
Read more05.10.17
The study was recently completed in May 2017. A total of 35 patients were enrolled across 12 sites in 8 countries. The primary objective of the 001 study was to characterize the individual patient’s disease progression profile through clinical, biological and quality-of-life measures recorded prospectively, as well as the historic disease information collected from patient medical records.
Read more20.09.17
There is an opportunity for you to participate in a market research study about Niemann-Pick Type C, conducted by an organization called ApotheCom on behalf of an industry sponsor. The goal of the study is to understand your preferences and concerns regarding the method through which a drug could be administered to manage Niemann-Pick Type C.
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