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.

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Niemann–Pick type C disease: cellular pathology and

Diagnostic workup and management of patients with suspected

Ocular motor biomarkers in Niemann-Pick disease type C: A

New formulation of FDA-approved drug may help treat Niemann-Pick

Niemann-Pick type C: contemporary diagnosis and treatment of a

Niemann-Pick disease type C clinical database: cognitive and

Frontiers Adult-Onset Niemann–Pick Disease Type C: Rapid

Niemann-Pick disease type C

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

Full article: New therapies in the management of Niemann-Pick type