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
Patterson, Marc C ; Mengel, Eugen ; Vanier, Marie T ; Schwierin, Barbara ; Muller, Audrey ; Cornelisse, Peter ; Pineda, Mercè
Orphanet journal of rare diseases, 2015-05, Vol.10 (1), p.65-65, Article 65 [Periódico revisado por pares]England: BioMed Central Ltd
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