Stable or improved neurological manifestations during miglustat therapy in patients from the international disease registry for Niemann-Pick disease type C: An observational cohort study

Marc C. Patterson, Eugen Mengel, Marie T. Vanier, Barbara Schwierin, Audrey Muller, Peter Cornelisse, Mercè Pineda, A. Amado-Fondo, Y. Amraoui, G. Andria, M. Arellano, B. Audoin, C. Azcona, C. Barr, J. Baruteau, C. Baumgartner, L. Bell, B. Bembi, K. Benneddif, G. BernardN. Bobocea, M. Bodzioch, T. Boettcher, M. Bonnan, P. Broue, A. Bruni, M. Caceres, R. Camino, E. Campbell, C. Cances, P. Cannell, J. Cesar, B. Chabrol, A. Chakrapani, R. Colao, A. Collet, T. Corsetti, A. Cousins, A. Covanis, T. Cox, J. M. Cuisset, A. Dardis, A. Das, P. Deegan, T. Dengler, F. Deodato, H. Derralynn, I. Di Donato, M. Di Rocco, G. Uziel, NPC Registry investigators

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