Potential treatment for Niemann-Pick type C, a rare neurodegenerative disease

Researchers in Japan have improved a potential treatment for a rare genetic disease, decreasing its negative toxic effects by threading it onto a dumb-bell-shaped chain and holding it in place until it reaches its target.

Niemann-Pick disease type C Alzheimer Society of Canada

Niemann-Pick Disease Concise Medical Knowledge

Lipid trafficking defects in Niemann-Pick type C disease

Psychiatric and Cognitive Symptoms Associated with Niemann-Pick

Myelin Defects in Niemann–Pick Type C Disease: Mechanisms and

Niemann–Pick Disease Type C: Induced Pluripotent Stem Cell–Derived

Niemann-Pick Type C Disease Reveals a Link between Lysosomal

Niemann-Pick Disease Type C

Combination therapy a potential strategy for treating Niemann-Pick

Molecular profile and peripheral markers of neurodegeneration in