A MULTICENTRE RETROSPECTIVE COHORT STUDY OF MIGLUSTAT IN PATIENTS WITH NIEMANN-PICK DISEASE TYPE C

MC Patterson, JF Wraith, E Mengel, F Sedel, W-L Hwu, M Rohrbach, B Bembi, M Walterfang, C Korenke, T Marquardt, C Luzy, R Giorgino, M Pineda