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Long-term neuropsychological outcomes from an open-label phase 1/2a trial of 2-hydoxypropyl-β-cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1

BACKGROUND: Niemann-Pick disease, type C1 is a neurodegenerative condition that arises from mutations of NPC1 and is often diagnosed in children. Recently, several drug trials have been implemented to minimize neurodegeneration, including a trial of 2-hydoxypropyl-β-cyclodextrins (VTS-270). OBJECTIV...

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Dettagli Bibliografici
Pubblicato in:CNS Drugs
Autori principali: Farmer, Cristan A., Thurm, Audrey, Farhat, Nicole, Bianconi, Simona, Keener, Lee Ann, Porter, Forbes D.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2019
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7448545/
https://ncbi.nlm.nih.gov/pubmed/31187454
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s40263-019-00642-2
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