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.

Treatment outcomes following continuous miglustat therapy in

Niemann-Pick disease type C

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Speaker Series - Niemann-Pick Disease Type C

University College London begins preclinical studies to develop a

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

Niemann Pick Disease - an overview

Efficacy and safety of arimoclomol in Niemann‐Pick disease type C

Rare Disease Video - Niemann-Pick Disease Types A & B - National

Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological

Recommendations for the detection and diagnosis of Niemann-Pick

Adult Niemann-Pick disease type C in France: clinical phenotypes

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

Niemann-Pick Type C Disease Reveals a Link between Lysosomal