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. The 001 study has already provided us with some valuable learnings: The NPC-CSS disease score were completed by patients with a good compliance, the skin biopsies and blood tests have been valuable for development and conformation of biomarkers, and we found that two-thirds of patients were treated with miglustat as part of their standard of care. Once all data have been analysed, we expect to gain insight about biomarkers and how the disease evolves over time in greater detail.Read more
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.Read more