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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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| Pubblicato in: | CNS Drugs |
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| Autori principali: | , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2019
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| Soggetti: | |
| 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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