Miglustat for treatment of Niemann-Pick C disease: a randomised
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Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect
New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Niemann-Pick disease type C
Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression
Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other
Miglustat, C10H21NO4
Niemann–Pick disease, type C - Wikipedia
Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult
Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study, Orphanet Journal of Rare Diseases
PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study
Orphazyme's arimoclomol to be used with Zavesca after FDA approval
Diagnostic workup and management of patients with suspected Niemann-Pick type C disease - Apostolos Papandreou, Paul Gissen, 2016
Miglustat in patients with Niemann-Pick disease Type C (NP-C): A
New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat
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