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Miglustat in patients with Niemann-Pick disease Type C (NP-C): A multicenter observational retrospective cohort study
M. Pineda, J. E. Wraith, E. Mengel, F. Sedel, W. L. Hwu, M. Rohrbach, B. Bembi, M. Walterfang, G. C. Korenke, T. Marquardt, C. Luzy, R. Giorgino, M. C. Patterson
Research output: Contribution to journal › Article › peer-review
144
Scopus
citations