Protocol for initiation of miglustat treatment in NP-C. Reproduced

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Gastrointestinal disturbances and their management in miglustat-treated patients

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Different Niemann-Pick C1 Genotypes Generate Protein Phenotypes that Vary in their Intracellular Processing, Trafficking and Localization

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Consensus clinical management guidelines for Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study, Orphanet Journal of Rare Diseases

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice

Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders - ScienceDirect

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

Protocol for initiation of miglustat treatment in NP-C. Reproduced with

Gastrointestinal disturbances and their management in miglustat‐treated patients - Belmatoug - 2011 - Journal of Inherited Metabolic Disease - Wiley Online Library

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

Frontiers Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases