{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,17]],"date-time":"2026-10-17T20:53:40Z","timestamp":1792270420827,"version":"4.4.0"},"reference-count":39,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2025,5,21]],"date-time":"2025-05-21T00:00:00Z","timestamp":1747785600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100002889","name":"Foundation for Prader-Willi Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002889","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J of Med Genetics Pt B"],"published-print":{"date-parts":[[2025,9]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>\n                    Prader\u2010Willi Syndrome (PWS), a rare genetic disorder, affects development and behavior, frequently resulting in self\u2010injury, aggression, hyperphagia, oppositional behavior, impulsivity, and over\u2010activity, causing significant morbidity. Currently, limited therapeutic options are available to manage these neuropsychiatric manifestations. A randomized, placebo\u2010controlled trial was conducted to assess the efficacy of guanfacine\u2010extended release (GXR) in reducing aggression and self\u2010injury in individuals with PWS. Subjects with a diagnosis of PWS, aged 6\u201335\u2009years with moderate to severe aggressive and\/or self\u2010injurious behavior, as determined by the clinical global impression (CGI)\u2010Severity scale, were included in an 8\u2010week double\u2010blind, placebo\u2010controlled, fixed\u2010flexible dose clinical trial of GXR, that was followed by an 8\u2010week open\u2010label extension phase. Validated behavioral instruments and physician assessments measured the efficacy of GXR treatment, its safety, and tolerability. GXR was effective in reducing aggression\/agitation and hyperactivity\/noncompliance, as measured by the Aberrant Behavior Checklist (ABC) scales (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009=\u20090.03). Overall aberrant behavior scores significantly reduced in the GXR arm. Aggression, as measured by the modified overt aggression scale (MOAS) also showed a significant reduction. Skin\u2010picking lesions, as measured by the self injury trauma (SIT) scale, decreased in response to GXR. No serious adverse events were experienced by any of the study participants. Fatigue\/sedation was the only adverse event significantly associated with GXR. The GXR group demonstrated significant overall clinical improvement, as measured by the CGI\u2010Improvement (CGI\u2010I) scale (\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.01). Findings of this pragmatic trial strongly support the use of GXR for the treatment of aggression, skin picking, and hyperactivity in children, adolescents, and adults with PWS.\n                  <\/jats:p>","DOI":"10.1002\/ajmg.b.33032","type":"journal-article","created":{"date-parts":[[2025,5,21]],"date-time":"2025-05-21T03:29:29Z","timestamp":1747798169000},"page":"62-70","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["A Randomized Double\u2010Blind Placebo\u2010Controlled Trial of Guanfacine Extended Release for Aggression and Self\u2010Injurious Behavior Associated With Prader\u2010Willi Syndrome"],"prefix":"10.1002","volume":"198","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1857-944X","authenticated-orcid":false,"given":"Deepan","family":"Singh","sequence":"first","affiliation":[{"name":"Department of Psychiatry Maimonides Medical Center  Brooklyn New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Silver","sequence":"additional","affiliation":[{"name":"Research Administration Maimonides Medical Center  Brooklyn New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Theresa","family":"Jacob","sequence":"additional","affiliation":[{"name":"Department of Psychiatry Maimonides Medical Center  Brooklyn New York USA"},{"name":"Clinical and Translational Research Labs Maimonides Medical Center  Brooklyn New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2025,5,21]]},"reference":[{"issue":"5","key":"e_1_2_10_2_1","first-page":"492","article-title":"Psychometric Characteristics of the Aberrant Behavior Checklist","volume":"89","author":"Aman M. 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P.","year":"2010","journal-title":"P T"},{"key":"e_1_2_10_9_1","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-012-0126-9"},{"key":"e_1_2_10_10_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.jcp.0000426184.68487.d3"},{"key":"e_1_2_10_11_1","doi-asserted-by":"publisher","DOI":"10.5664\/jcsm.9938"},{"key":"e_1_2_10_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2788.2005.00726.x"},{"key":"e_1_2_10_13_1","first-page":"245","article-title":"Self\u2010Injurious Behavior and Serotonin in Prader\u2010Willi Syndrome","volume":"20","author":"Hellings J. A.","year":"1994","journal-title":"Psychopharmacology Bulletin"},{"key":"e_1_2_10_14_1","doi-asserted-by":"publisher","DOI":"10.1097\/chi.0b013e318068fbed"},{"key":"e_1_2_10_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.euroneuro.2014.09.014"},{"key":"e_1_2_10_16_1","doi-asserted-by":"publisher","DOI":"10.1901\/jaba.1990.23-99"},{"key":"e_1_2_10_17_1","doi-asserted-by":"publisher","DOI":"10.1097\/WNF.0000000000000262"},{"key":"e_1_2_10_18_1","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0194655"},{"key":"e_1_2_10_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/eat.1062"},{"key":"e_1_2_10_20_1","doi-asserted-by":"publisher","DOI":"10.1186\/1471-244X-11-173"},{"key":"e_1_2_10_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/cen.13169"},{"key":"e_1_2_10_22_1","doi-asserted-by":"publisher","DOI":"10.5664\/jcsm.7258"},{"key":"e_1_2_10_23_1","unstructured":"Matesevac L.2021.\u201cPATH for PWS Study 2021 Update: A Non\u2010Interventional Observational Natural History Study of Serious Medical Events in Prader\u2010Willi Syndrome.\u201d Paper Presented at in the PWSA\u2010USA Medical & Scientific Conference 2021 Virtual.https:\/\/www.pwsausa.org\/2021\u2010national\u2010convention\u2010session\u2010recordings\/."},{"key":"e_1_2_10_24_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-023-48024-5"},{"key":"e_1_2_10_25_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.36306"},{"key":"e_1_2_10_26_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.38160"},{"key":"e_1_2_10_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.a.36615"},{"key":"e_1_2_10_28_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.braindev.2007.12.011"},{"key":"e_1_2_10_29_1","doi-asserted-by":"publisher","DOI":"10.1177\/070674371506000202"},{"key":"e_1_2_10_30_1","doi-asserted-by":"publisher","DOI":"10.1176\/appi.ajp.2015.15010055"},{"key":"e_1_2_10_31_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00213-012-2893-8"},{"key":"e_1_2_10_32_1","doi-asserted-by":"publisher","DOI":"10.1089\/cap.2014.0141"},{"key":"e_1_2_10_33_1","doi-asserted-by":"publisher","DOI":"10.1089\/cap.2018.0102"},{"key":"e_1_2_10_34_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2788.2006.00895.x"},{"key":"e_1_2_10_35_1","doi-asserted-by":"publisher","DOI":"10.1186\/1750-1172-6-47"},{"key":"e_1_2_10_36_1","doi-asserted-by":"publisher","DOI":"10.1111\/jir.12010"},{"key":"e_1_2_10_37_1","doi-asserted-by":"publisher","DOI":"10.1001\/archderm.1992.04530010061009"},{"key":"e_1_2_10_38_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2788.2005.00690.x"},{"key":"e_1_2_10_39_1","unstructured":"Drugs@FDA: FDA Approved Drug Products. n.d.\u201cAccessdata.fda.gov.\u201dwww.accessdata.fda.gov\/scripts\/cder\/daf\/index.cfm?event=overview.process&applno=200881."},{"key":"e_1_2_10_40_1","doi-asserted-by":"publisher","DOI":"10.1176\/ajp.143.1.35"}],"container-title":["American Journal of Medical Genetics Part B: Neuropsychiatric Genetics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/ajmg.b.33032","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,8,9]],"date-time":"2025-08-09T10:15:35Z","timestamp":1754734535000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/ajmg.b.33032"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,5,21]]},"references-count":39,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["10.1002\/ajmg.b.33032"],"URL":"https:\/\/doi.org\/10.1002\/ajmg.b.33032","archive":["Portico"],"relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2024.08.22.24312419","asserted-by":"object"}]},"ISSN":["1552-4841","1552-485X"],"issn-type":[{"value":"1552-4841","type":"print"},{"value":"1552-485X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,5,21]]},"assertion":[{"value":"2025-01-24","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-04-25","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-05-21","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}