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

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

PDF) Goal-oriented therapy with miglustat in Gaucher disease

PDF) Goal-oriented therapy with miglustat in Gaucher disease

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

PDF) Effects of miglustat therapy on neurological disorder and survival in early-infantile Niemann-Pick disease type C: a national French retrospective study

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

A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus - Rasmussen - 2023 - Journal of Neurochemistry - Wiley Online Library

Elucidating the mechanism of cyclodextrins in the treatment of Niemann-Pick Disease Type C using crosslinked 2-hydroxypropyl-β-cyclodextrin

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

Imaging of neuroinflammation in adult Niemann-Pick type C disease

Niemann-Pick disease treatment: a systematic review of clinical trials.

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

Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin. - Abstract - Europe PMC