Individual clinical data: SF-36, EDSS and ALDS values. SF-36 values were compared with norm values for the Dutch population and corrected for gender and age. The SF-36 subdomain scores are expressed i
The ERICA Patient Reported Outcome Measures (PROMs) Repository is the first attempt to identify and centralize Clinical Assessment Outcomes questionnaires of relevance for rare diseases and constitute
Niemann-Pick Disease, type C (NPC) is a fatal, neurodegenerative, lysosomal storage disorder. It is a rare disease with broad phenotypic spectrum and variable age of onset. These issues make it diffic
