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

PDF) Goal-oriented therapy with miglustat in Gaucher disease

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

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

Treatment outcomes following continuous miglustat therapy in patients with Niemann-Pick disease Type C: a final report of the NPC Registry, Orphanet Journal of Rare Diseases

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

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

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

New murine Niemann-Pick type C models bearing a pseudoexon-generating mutation recapitulate the main neurobehavioural and molecular features of the disease

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

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

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

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

Estimated prevalence of Niemann–Pick type C disease in Quebec

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