Orphanet Journal of Rare Diseases | Abstract | 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

Orphanet Journal of Rare Diseases | Abstract | 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

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Research  

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 MC, Mengel E, Vanier MT, Schwierin B, Muller A, Cornelisse P, Pineda M, On behalf of the NPC Registry investigators

Orphanet Journal of Rare Diseases 2015, 10 :65 (28 May 2015)

Abstract | Provisional PDF