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[cited 2020 Apr 21]. Available from: https:\/\/www.streetinsider.com\/Corporate+News\/Prana+Biotech+%28PRAN%29+Announces+Partial+Clinical+Hold+on+PBT2\/10265824.html. 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Huntingtin suppression restores cognitive function in a mouse model of Huntington's disease. Sci Transl Med [Internet]. 2018 Oct 3 [cited 2020 Apr 3];10(461). 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[cited 2020 Apr 12]. Available from: https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03810898. Accessed 1 May 2020"},{"issue":"3","key":"10.1007\/s13311-020-00891-w_bib142","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1016\/j.parkreldis.2008.05.012","article-title":"Levels of the light subunit of neurofilament triplet protein in cerebrospinal fluid in Huntington's disease","volume":"15","author":"Constantinescu","year":"2009","journal-title":"Parkinsonism Relat Disord."},{"issue":"1","key":"10.1007\/s13311-020-00891-w_bib143","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1111\/jnc.13719","article-title":"Cerebrospinal fluid total tau concentration predicts clinical phenotype in Huntington's disease","volume":"139","author":"Rodrigues","year":"2016","journal-title":"J Neurochem."},{"issue":"8","key":"10.1007\/s13311-020-00891-w_bib144","doi-asserted-by":"crossref","first-page":"601","DOI":"10.1016\/S1474-4422(17)30124-2","article-title":"Neurofilament light protein in blood as a potential biomarker of neurodegeneration in Huntington's disease: a retrospective cohort analysis","volume":"16","author":"Byrne","year":"2017","journal-title":"Lancet Neurol."}],"container-title":["Neurotherapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s13311-020-00891-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s13311-020-00891-w\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747923012631?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1878747923012631?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s13311-020-00891-w.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,30]],"date-time":"2025-10-30T06:59:11Z","timestamp":1761807551000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1878747923012631"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,10]]},"references-count":144,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2020,10]]}},"alternative-id":["S1878747923012631"],"URL":"https:\/\/doi.org\/10.1007\/s13311-020-00891-w","relation":{},"ISSN":["1878-7479"],"issn-type":[{"value":"1878-7479","type":"print"}],"subject":[],"published":{"date-parts":[[2020,10]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Therapeutic Update on Huntington's Disease: Symptomatic Treatments and Emerging Disease-Modifying Therapies","name":"articletitle","label":"Article Title"},{"value":"Neurotherapeutics","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1007\/s13311-020-00891-w","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2020 The American Society for Experimental NeuroTherapeutics, Inc. 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