{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,10]],"date-time":"2026-11-10T01:35:50Z","timestamp":1794274550800,"version":"4.9.0"},"reference-count":38,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2024,1,1]],"date-time":"2024-01-01T00:00:00Z","timestamp":1704067200000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100015661","name":"UCB Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015661","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ther Adv Neurol Disord"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Generalized myasthenia gravis (gMG) is a chronic, unpredictable disease associated with high treatment and disease burdens, with a need for more effective and well-tolerated treatments.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>To evaluate the long-term safety, tolerability, and efficacy of zilucoplan in a mild-to-severe, acetylcholine receptor autoantibody-positive (AChR+) gMG population.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>Ongoing, multicenter, phase III open-label extension (OLE) study.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Eligible patients had completed a qualifying randomized, placebo-controlled phase II or phase III zilucoplan study and received daily, self-administered subcutaneous 0.3\u2009mg\/kg zilucoplan. The primary endpoint was incidence of treatment-emergent adverse events (TEAEs). Secondary efficacy endpoints included change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>In total, 200 patients enrolled. At the cut-off date (8 September 2022), median (range) exposure to zilucoplan in RAISE-XT was 1.2 (0.11\u20134.45)\u2009years. Mean age at OLE baseline was 53.3\u2009years. A total of 188 (94%) patients experienced a TEAE, with the most common being MG worsening ( n\u2009=\u200952, 26%) and COVID-19 ( n\u2009=\u200949, 25%). In patients who received zilucoplan 0.3\u2009mg\/kg in the parent study, further improvements in MG-ADL score continued through to Week 24 (least squares mean change [95% confidence interval] from double-blind baseline \u22126.06 [\u22127.09, \u22125.03]) and were sustained through to Week 60 (\u22126.04 [\u22127.21, \u22124.87]). In patients who switched from placebo in the parent study, rapid improvements in MG-ADL score were observed at the first week after switching to zilucoplan; further improvements were observed at Week 24, 12\u2009weeks after switching (\u22126.46 [\u22128.19, \u22124.72]), and were sustained through to Week 60 (\u22126.51 [\u22128.37, \u22124.65]). Consistent results were observed in other efficacy endpoints.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Zilucoplan demonstrated a favorable long-term safety profile, good tolerability, and sustained efficacy through to Week 60 with consistent benefits in a broad AChR+ gMG population. Additional long-term data will be available in future analyses.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration:<\/jats:title>\n                    <jats:p>ClinicalTrials.gov identifier: NCT04225871 ( https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04225871 )<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/17562864241243186","type":"journal-article","created":{"date-parts":[[2024,4,17]],"date-time":"2024-04-17T07:56:25Z","timestamp":1713340585000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":54,"title":["Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7136-8617","authenticated-orcid":false,"suffix":"Jr","given":"James F.","family":"Howard","sequence":"first","affiliation":[{"name":"Department of Neurology, UNC School of Medicine, The University College of North Carolina at Chapel Hill, 2200 Houpt Building, CB#7025, 170 Manning Drive, Chapel Hill, NC 27599-7025, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saskia","family":"Bresch","sequence":"additional","affiliation":[{"name":"Service de Neurologie, Hospital Pasteur, Centre Hospitalier Universitaire de Nice, Nice, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Constantine","family":"Farmakidis","sequence":"additional","affiliation":[{"name":"Neuromuscular Division, Department of Neurology, University of Kansas Medical Center, Kansas City, KS, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miriam","family":"Freimer","sequence":"additional","affiliation":[{"name":"Department of Neurology, The Ohio State University Wexner Medical Center, Columbus, OH, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Genge","sequence":"additional","affiliation":[{"name":"Clinical Research Unit, Montreal Neurological Institute, Montreal, QC, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Channa","family":"Hewamadduma","sequence":"additional","affiliation":[{"name":"Academic Neuroscience Unit, Sheffield Teaching Hospitals Foundation Trust, Sheffield, UK"},{"name":"Sheffield Institute for Translational Neurosciences (SITRAN), University of Sheffield, Sheffield, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Hinton","sequence":"additional","affiliation":[{"name":"Department of Neurology, Frederick P. Whiddon School of Medicine, University of South Alabama, Mobile, AL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yessar","family":"Hussain","sequence":"additional","affiliation":[{"name":"Department of Neurology, Dell Medical School, The University of Texas at Austin, Austin, TX, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raul","family":"Juntas-Morales","sequence":"additional","affiliation":[{"name":"Department of Neurology, Vall d\u2019Hebron University Hospital, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Henry J.","family":"Kaminski","sequence":"additional","affiliation":[{"name":"Department of Neurology and Rehabilitation Medicine, George Washington University, Washington, DC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angelina","family":"Maniaol","sequence":"additional","affiliation":[{"name":"Department of Neurology, Oslo University Hospital, Oslo, Norway"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9810-5737","authenticated-orcid":false,"given":"Renato","family":"Mantegazza","sequence":"additional","affiliation":[{"name":"Fondazione Istituto di Ricovero e Cura a Carattere Scientifico, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masayuki","family":"Masuda","sequence":"additional","affiliation":[{"name":"Department of Neurology, Tokyo Medical University, Tokyo, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard J.","family":"Nowak","sequence":"additional","affiliation":[{"name":"Department of Neurology, Yale School of Medicine, New Haven, CT, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kumaraswamy","family":"Sivakumar","sequence":"additional","affiliation":[{"name":"Neuromuscular Clinic and Research Center, Phoenix, AZ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marek","family":"\u015ami\u0142owski","sequence":"additional","affiliation":[{"name":"Department of Hematology and Bone Marrow Transplantation, Medical University of Silesia, Katowice, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kimiaki","family":"Utsugisawa","sequence":"additional","affiliation":[{"name":"Department of Neurology, Hanamaki General Hospital, Hanamaki, Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tuan","family":"Vu","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of South Florida Morsani College of Medicine, Tampa, FL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael D.","family":"Weiss","sequence":"additional","affiliation":[{"name":"Department of Neurology, University of Washington Medical Center, Seattle, WA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ma\u0142gorzata","family":"Zajda","sequence":"additional","affiliation":[{"name":"Department of Experimental and Clinical Pharmacology, Medical University of Warsaw, Warsaw, Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jos","family":"Bloemers","sequence":"additional","affiliation":[{"name":"UCB Pharma, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Babak","family":"Boroojerdi","sequence":"additional","affiliation":[{"name":"UCB Pharma, Monheim am Rhein, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Brock","sequence":"additional","affiliation":[{"name":"UCB Pharma, Raleigh, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-6483-0925","authenticated-orcid":false,"given":"Guillemette","family":"de la Borderie","sequence":"additional","affiliation":[{"name":"UCB Pharma, Brussels, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Petra W.","family":"Duda","sequence":"additional","affiliation":[{"name":"UCB Pharma, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark","family":"Vanderkelen","sequence":"additional","affiliation":[{"name":"UCB Pharma, Braine-L\u2019Alleud, Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M. 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