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

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

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases

Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C, Orphanet Journal of Rare Diseases

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

IJMS, Free Full-Text

Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice: Molecular Therapy

NPC1 enables cholesterol mobilization during long‐term potentiation that can be restored in Niemann–Pick disease type C by CYP46A1 activation

Adult Niemann-Pick disease type C in France: clinical phenotypes and long-term miglustat treatment effect, Orphanet Journal of Rare Diseases

Full article: New therapies in the management of Niemann-Pick type C disease: clinical utility of miglustat

Miglustat - wikidoc

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