{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T05:48:55Z","timestamp":1788241735742,"version":"build-2803163510"},"reference-count":12,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2025,1,4]],"date-time":"2025-01-04T00:00:00Z","timestamp":1735948800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100006259","name":"Teva Pharmaceutical Industries","doi-asserted-by":"publisher","award":["TV50717\u2010NDG\u201060301"],"award-info":[{"award-number":["TV50717\u2010NDG\u201060301"]}],"id":[{"id":"10.13039\/100006259","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["movementdisorders.onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Movement Disord Clin Pract"],"published-print":{"date-parts":[[2025,4]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Dystonia may respond to VMAT2 inhibition.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Providing pilot data on the safety, tolerability, and efficacy of deutetrabenazine in non dopa\u2010responsive dystonia.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Deutetrabenazine was titrated by adults with isolated dystonia. Primary study endpoints included the proportion who maintained the individual, maximum tolerated dose for 6\u2009weeks, and how many titrated to 48\u2009mg\/day. Secondary endpoints included rates of QTc prolongation\/arrhythmias, suicidality, excessive daytime sleepiness, cognitive decline, and drug\u2010induced parkinsonism. Exploratory endpoints for clinical efficacy were assessed.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 15 participants, four (26.7%) withdrew early and six (40%) titrated to 48\u2009mg\/day. Common adverse events included fatigue and diarrhea. Secondary safety endpoints did not change significantly, but MDS\u2010UPDRS III scores worsened by \u22653 points in seven participants (46.7%). PGI\u2010C and the blinded CGI\u2010C and GDS improved in three women with blepharospasm.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Most participants tolerated deutetrabenazine for 6\u2009weeks, and those with blepharospasm may have benefitted from its use.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mdc3.14327","type":"journal-article","created":{"date-parts":[[2025,1,4]],"date-time":"2025-01-04T03:18:48Z","timestamp":1735960728000},"page":"504-509","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["An Open\u2010Label Pilot Study to Examine the Safety, Tolerability and Efficacy of Deutetrabenazine in Isolated Dystonia"],"prefix":"10.1002","volume":"12","author":[{"given":"Andres","family":"Deik","sequence":"first","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8130-2809","authenticated-orcid":false,"given":"Whitley","family":"Aamodt","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina","family":"Cadet","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aaron","family":"Lasker","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexandria","family":"Oliver","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Meredith","family":"Spindler","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas F.","family":"Tropea","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1826-6275","authenticated-orcid":false,"given":"Pavan","family":"Vaswani","sequence":"additional","affiliation":[{"name":"Parkinson Disease and Movement Disorders Center University of Pennsylvania  Philadelphia Pennsylvania 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