Miglustat for treatment of Niemann-Pick C disease: a randomised
Por um escritor misterioso
Last updated 22 novembro 2024
The pathogenesis of Niemann–Pick type C disease: a role for autophagy?, Expert Reviews in Molecular Medicine
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
Imaging of neuroinflammation in adult Niemann-Pick type C disease
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
Niemann-Pick Disease Drug Type C Treatment Market Size
IJMS, Free Full-Text
Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases
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