{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,21]],"date-time":"2025-10-21T00:43:58Z","timestamp":1761007438590,"version":"build-2065373602"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,3,4]],"date-time":"2020-03-04T00:00:00Z","timestamp":1583280000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000071","name":"National Institute of Child Health and Human Development","doi-asserted-by":"publisher","award":["HD036071"],"award-info":[{"award-number":["HD036071"]}],"id":[{"id":"10.13039\/100000071","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":[[2020,4]]},"abstract":"<jats:title>ABSTRACT<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Fragile X\u2010associated tremor\/ataxia syndrome (FXTAS) is a neurodegenerative disorder characterized by intention tremor, cerebellar ataxia, and executive dysfunction in carriers of a CGG repeat expansion premutation (55\u2013200 repeats) in the fragile X mental retardation 1 (<jats:italic>FMR1<\/jats:italic>) gene. Given reports of poor insight in FXTAS, we postulated that patients with FXTAS would be less likely to exhibit placebo response.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>To analyze placebo response from the first randomized controlled trial in FXTAS that evaluated cognitive and motor outcomes after 1 year of treatment with memantine.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Data from the placebo arm of the first randomized controlled trial in FXTAS were analyzed. There were 2 coprimary outcomes. Based on studies in Parkinson's disease, placebo responders were defined as individuals with an improvement of at least 50% in the coprimary outcomes. Improvements of 20% and 30% served as secondary cutoff values based on the suggested magnitude of placebo response in other movement disorders.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 36 participants in the placebo group completed baseline and follow\u2010up evaluations. The average age was 66\u2009\u00b1\u20097\u2009years, and 60% were men. Average CGG repeat size was 86\u2009\u00b1\u200918. A total of 19 participants had stage 3 disease. Only 1 patient showed 50% improvement in both coprimary outcomes. At 30% and 20% improvement, there were 2 and 3 patients showing placebo response in the coprimary outcomes, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Patients with FXTAS exhibited low rates of placebo response in a randomized controlled trial. Further studies on the relationship between baseline insight and placebo responsivity are applicable to FXTAS and other disorders exhibiting cognitive impairment.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mdc3.12919","type":"journal-article","created":{"date-parts":[[2020,2,22]],"date-time":"2020-02-22T08:14:35Z","timestamp":1582359275000},"page":"298-302","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Placebo Response in Fragile X\u2010associated Tremor\/Ataxia Syndrome"],"prefix":"10.1002","volume":"7","author":[{"given":"Emily J.","family":"Hill","sequence":"first","affiliation":[{"name":"Department of Neurology Baylor College of Medicine  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christopher G.","family":"Goetz","sequence":"additional","affiliation":[{"name":"Department of Neurological Sciences Rush University Medical Center  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Glenn T.","family":"Stebbins","sequence":"additional","affiliation":[{"name":"Department of Neurological Sciences Rush University Medical Center  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Randi","family":"Hagerman","sequence":"additional","affiliation":[{"name":"University of California Davis Medical Investigation of Neurodevelopmental Disorders (MIND) Institute  Sacramento California USA"},{"name":"Department of Pediatrics University of California Davis Medical Center  Sacramento California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bichun","family":"Ouyang","sequence":"additional","affiliation":[{"name":"Department of Neurological Sciences Rush University Medical Center  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Deborah A.","family":"Hall","sequence":"additional","affiliation":[{"name":"Department of Neurological Sciences Rush University Medical Center  Chicago Illinois USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2020,3,4]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.57.1.127"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1086\/374321"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.21252"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1159\/000441566"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.3389\/fgene.2014.00365"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.2147\/CIA.S1794"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.3233\/JAD-170672"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10803-009-0807-3"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1097\/JCP.0b013e3181f1d10a"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.4088\/JCP.13m08546"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(16)00066-1"},{"key":"e_1_2_8_13_1","unstructured":"HallD GrigsbyJ HagermanR et al. Discordant impairment perceptions in FXTAS: patients vs. experienced raters. In:10th International Congress of Parkinson's Disease and Movement Disorders. Kyoto Japan:Movement Disorders2006;21 Suppl. 15:S331\u2010S332."},{"key":"e_1_2_8_14_1","doi-asserted-by":"publisher","DOI":"10.1093\/arclin\/acu022"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1096\/fj.201600315R"},{"key":"e_1_2_8_16_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.54.3.710"},{"key":"e_1_2_8_17_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.26580"},{"key":"e_1_2_8_18_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.27416"},{"key":"e_1_2_8_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.24062"},{"key":"e_1_2_8_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.25365"},{"key":"e_1_2_8_21_1","doi-asserted-by":"publisher","DOI":"10.1080\/13854046.2016.1185100"},{"key":"e_1_2_8_22_1","doi-asserted-by":"publisher","DOI":"10.1080\/13854040701336436"},{"key":"e_1_2_8_23_1","doi-asserted-by":"publisher","DOI":"10.1111\/pcn.12006"},{"key":"e_1_2_8_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ctim.2012.06.002"},{"key":"e_1_2_8_25_1","doi-asserted-by":"publisher","DOI":"10.1037\/0894-4105.22.1.48"},{"key":"e_1_2_8_26_1","doi-asserted-by":"publisher","DOI":"10.1093\/arclin\/18.2.153"},{"key":"e_1_2_8_27_1","doi-asserted-by":"publisher","DOI":"10.1186\/1471-2202-11-118"},{"key":"e_1_2_8_28_1","doi-asserted-by":"publisher","DOI":"10.3389\/fpsyg.2017.01231"},{"key":"e_1_2_8_29_1","doi-asserted-by":"publisher","DOI":"10.1002\/mdc3.12708"},{"key":"e_1_2_8_30_1","doi-asserted-by":"publisher","DOI":"10.1002\/mds.21894"}],"container-title":["Movement Disorders Clinical Practice"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmdc3.12919","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mdc3.12919","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mdc3.12919","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/movementdisorders.onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mdc3.12919","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T13:21:15Z","timestamp":1760966475000},"score":1,"resource":{"primary":{"URL":"https:\/\/movementdisorders.onlinelibrary.wiley.com\/doi\/10.1002\/mdc3.12919"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,3,4]]},"references-count":29,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2020,4]]}},"alternative-id":["10.1002\/mdc3.12919"],"URL":"https:\/\/doi.org\/10.1002\/mdc3.12919","archive":["Portico"],"relation":{},"ISSN":["2330-1619","2330-1619"],"issn-type":[{"type":"print","value":"2330-1619"},{"type":"electronic","value":"2330-1619"}],"subject":[],"published":{"date-parts":[[2020,3,4]]},"assertion":[{"value":"2019-11-21","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-02-20","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-03-04","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}