Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: A Prospective Non-therapeutic Study in Patients Diagnosed With Niemann-Pick Disease Type C in Order to Characterise the Individual Patient Disease Profile and Historic Sig
Brief Summary:
This is a prospective non-therapeutic observational study in NP-C patients. The aim is to characterize the individual patient disease progression profile through the historical and 6 months prospective evaluation of clinical, imaging, biological(biomarkers) and quality of life data.
Patients will be offered enrollment into a Phase II/III study on arimoclomol at the end of the study.