Selected Articles
- Niemann-Pick disease type C
- A novel, highly sensitive and specific biomarker for Niemann-Pick type C1 disease
- P352 - Use of Cyclodextrin in two Brazilian Girls with Niemann-Pick Disease Type C
- High Incidence of Unrecognized Visceral/Neurological Late- onset Niemann-Pick Disease, type C1 Predicted by Analysis of Massively Parallel Sequencing Data Sets
- Linear Clinical Progression, Independent of Age of Onset, in Niemann-Pick Disease, type C
- Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood–brain barrier permeability
- Pulmonary involvement in Niemann-Pick C type 1
- Niemann-Pick C disease and mobilization of lysosomal cholesterol by cyclodextrin
- Neuroprotection by cyclodextrin in cell and mouse models of Alzheimer disease
- Therapeutic potential of cyclodextrins in the treatment of Niemann–Pick type C disease
- Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-/- mouse
- Cyclodextrins as Emerging Therapeutic Tools in the Treatment of Cholesterol-Associated Vascular and Neurodegenerative Diseases
