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

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

Loss of NPC1 enhances phagocytic uptake and impairs lipid trafficking in microglia

Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice

Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late- Onset Pompe Disease

PDF) Goal-oriented therapy with miglustat in Gaucher disease

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

Oxidative stress: a pathogenic mechanism for Niemann-Pick type C disease. - Abstract - Europe PMC

Phenotypic expression of swallowing function in Niemann–Pick disease type C1, Orphanet Journal of Rare Diseases

Severely impaired CTL killing is a feature of the neurological disorder Niemann-Pick disease type C1 - ScienceDirect

Impact of imiglucerase supply constraint on the therapeutic management and course of disease in French patients with Gaucher disease type 1 – topic of research paper in Clinical medicine. Download scholarly article