Searching for a cure for Niemann-Pick Type C - NemaMetrix

Niemann-Pick Type C is a rare and fatal disorder affecting children. We explore the efficacy of new potential therapeutics with the ScreenChip System.

Molecular profile and peripheral markers of neurodegeneration in patients with Niemann-Pick type C: Decrease in Plasminogen Activator Inhibitor type 1 and Platelet-Derived Growth Factor type AA - ScienceDirect

PDF) Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-Β-Cyclodextrin

Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study - The Lancet Neurology

PDF) Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-Β-Cyclodextrin

Investigation of Brain Iron in Niemann-Pick Type C: A 7T Quantitative Susceptibility Mapping Study

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C

Recent advances in the treatment of Niemann pick disease type C: A mini-review - ScienceDirect

Intrathecal cyclodextrin in the treatment of Niemann-Pick disease type C

Imaging of neuroinflammation in adult Niemann-Pick type C disease