Long-Term Treatment of Niemann-Pick Type C1 Disease with Intrathecal 2-Hydroxypropyl-β-Cyclodextrin

BACKGROUND: Intrathecal 2-hydoxypropyl-β-cyclodextrin (HPβCD) has been found to mobilize cholesterol, extend life, reduce cerebellar pathology and delay onset of ataxia in the mouse and cat models of Niemann-Pick disease, type C1 (NPC1), a clinically variable progressive and ultimately fatal neurode...

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Publicat a:Pediatr Neurol
Autors principals: Berry-Kravis, Elizabeth, Chin, Jamie, Hoffmann, Anne, Winston, Amy, Stoner, Robin, LaGorio, Lisa, Friedmann, Katherine, Hernandez, Mariana, Ory, Daniel S., Porter, Forbes D, O’Keefe, Joan A.
Format: Artigo
Idioma:Inglês
Publicat: 2018
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Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5857219/
https://ncbi.nlm.nih.gov/pubmed/29429782
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.pediatrneurol.2017.12.014
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