{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T19:19:40Z","timestamp":1791314380275,"version":"4.3.1"},"reference-count":13,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2017,4,24]],"date-time":"2017-04-24T00:00:00Z","timestamp":1492992000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"French Ministry of Health","award":["PHRC 2004-03bis CYST-HD"],"award-info":[{"award-number":["PHRC 2004-03bis CYST-HD"]}]},{"name":"Raptor Pharmaceuticals Inc. (USA)"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Movement Disorders"],"published-print":{"date-parts":[[2017,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:p><jats:bold>Background<\/jats:bold>: Cysteamine has been demonstrated as potentially effective in numerous animal models of Huntington's disease.<\/jats:p><jats:p><jats:bold>Methods<\/jats:bold>: Ninety\u2010six patients with early\u2010stage Huntington's disease were randomized to 1200\u2009mg delayed\u2010release cysteamine bitartrate or placebo daily for 18 months. The primary end point was the change from baseline in the UHDRS Total Motor Score. A linear mixed\u2010effects model for repeated measures was used to assess treatment effect, expressed as the least\u2010squares mean difference of cysteamine minus placebo, with negative values indicating less deterioration relative to placebo.<\/jats:p><jats:p><jats:bold>Results<\/jats:bold>: At 18 months, the treatment effect was not statistically significant \u2014 least\u2010squares mean difference, \u20101.5\u2009\u00b1\u20091.71 (<jats:italic>P<\/jats:italic>\u2009=\u20090.385) \u2014 although this did represent less mean deterioration from baseline for the treated group relative to placebo. Treatment with cysteamine was safe and well tolerated.<\/jats:p><jats:p><jats:bold>Conclusions<\/jats:bold>: Efficacy of cysteamine was not demonstrated in this study population of patients with Huntington's disease. Post hoc analyses indicate the need for definitive future studies. \u00a9 2017 International Parkinson and Movement Disorder Society<\/jats:p>","DOI":"10.1002\/mds.27010","type":"journal-article","created":{"date-parts":[[2017,4,24]],"date-time":"2017-04-24T06:06:46Z","timestamp":1493014006000},"page":"932-936","source":"Crossref","is-referenced-by-count":45,"title":["A randomized, double\u2010blind, placebo\u2010controlled trial evaluating cysteamine in Huntington's disease"],"prefix":"10.1002","volume":"32","author":[{"given":"Christophe","family":"Verny","sequence":"first","affiliation":[{"name":"Centre Hospitalier Universitaire d'Angers, D\u00e9partement de Neurologie et UMR CNRS 6214 \u2013 INSERM U1083 et Institut Mitovasc Angers France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne\u2010Catherine","family":"Bachoud\u2010L\u00e9vi","sequence":"additional","affiliation":[{"name":"Assistance Publique\u2010H\u00f4pitaux de Paris Centre National de R\u00e9f\u00e9rence Maladie de Huntington, Centre Hospitalier Universitaire H. Mondor \u2014 A. Chenevier de Cr\u00e9teil et INSERM U955, Equipe 01 Neuropsychologie interventionnelle, Cr\u00e9teil et Ecole Normale Sup\u00e9rieure, Institut d'Etudes Cognitives, Paris et Universit\u00e9 Paris\u2010Est, Facult\u00e9 de M\u00e9decine Cr\u00e9teil France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandra","family":"Durr","sequence":"additional","affiliation":[{"name":"Assistance Publique\u2010H\u00f4pitaux de Paris D\u00e9partement de G\u00e9n\u00e9tique, and Institut du Cerveau et de la Moelle \u00e9pini\u00e8re, H\u00f4pital de la Piti\u00e9\u2010Salp\u00eatri\u00e8re Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cyril","family":"Goizet","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire de Bordeaux H\u00f4pital Pellegrin, Service de G\u00e9n\u00e9tique M\u00e9dicale, Universit\u00e9 de Bordeaux, INSERM U1211 Bordeaux France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean\u2010Philippe","family":"Azulay","sequence":"additional","affiliation":[{"name":"Assistance Publique\u2010H\u00f4pitaux de Marseille H\u00f4pital de la Timone, D\u00e9partement de neurologie et de pathologie du mouvement, Institut de neurosciences de la Timone UMR 7289 AMU\u2010CNRS Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cl\u00e9mence","family":"Simonin","sequence":"additional","affiliation":[{"name":"Institut de Recherche sur le Cancer de Lille, INSERM UMR837, Centre Hospitalier Universitaire de Lille, D\u00e9partement de Neurologie et des Mouvements Anormaux Lille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine","family":"Tranchant","sequence":"additional","affiliation":[{"name":"H\u00f4pitaux Universitaire de Strasbourg H\u00f4pital Hautepierre, Service de Neurologie, Unit\u00e9 des Pathologies du mouvement Strasbourg France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fabienne","family":"Calvas","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire Purpan, Centre d'Investigation Clinique Toulouse France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Krystkowiak","sequence":"additional","affiliation":[{"name":"Centre Hospitalier Universitaire d'Amiens D\u00e9partement de Neurologie, Universit\u00e9 de Picardie Jules Verne, EA4559, Laboratoire de Neurosciences Fonctionnelles et Pathologie Amiens France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Perrine","family":"Charles","sequence":"additional","affiliation":[{"name":"Assistance Publique\u2010H\u00f4pitaux de Paris D\u00e9partement de G\u00e9n\u00e9tique, and Institut du Cerveau et de la Moelle \u00e9pini\u00e8re, H\u00f4pital de la Piti\u00e9\u2010Salp\u00eatri\u00e8re Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katia","family":"Youssov","sequence":"additional","affiliation":[{"name":"Assistance Publique\u2010H\u00f4pitaux de Paris Centre National de R\u00e9f\u00e9rence Maladie de Huntington, Centre Hospitalier Universitaire H. 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