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, NPC Registry investigators

Research output: Contribution to journal › Article › peer-review

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Medicine and Dentistry