Miglustat for treatment of Niemann-Pick C disease: a randomised

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Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid Treatment Initiation Advised but Early Diagnosis Remains Difficult

Necroptosis in Niemann–Pick disease, type C1: a potential therapeutic target

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C: Results from a double‐blind, randomised, placebo‐controlled, multinational phase 2/3 trial of a novel treatment - Mengel - 2021 - Journal of Inherited

Prevalence of dysphagia in Niemann-Pick disease type C (NP-C) and other

Correlation of age of onset and clinical severity in Niemann–Pick disease type C1 with lysosomal abnormalities and gene expression

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Finding pathogenic commonalities between Niemann-Pick type C and other lysosomal storage disorders: Opportunities for shared therapeutic interventions - ScienceDirect

PDF] Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study

PDF) Recommendations on the diagnosis and management of Niemann-Pick disease type C

Miglustat in patients with Niemann-Pick disease Type C (NP-C): A

Niemann–Pick disease, type C - Wikipedia

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IJMS, Free Full-Text

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases