{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T09:31:24Z","timestamp":1793266284389,"version":"4.5.0"},"reference-count":37,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T00:00:00Z","timestamp":1774742400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T00:00:00Z","timestamp":1774742400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/501100006586","name":"Centre Hospitalier Universitaire de Nice","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100006586","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"published-print":{"date-parts":[[2026,11]]},"abstract":"<jats:sec>\n                    <jats:title>Objectives<\/jats:title>\n                    <jats:p>To evaluate the effectiveness and safety of zilucoplan in the real-world treatment setting in France.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This retrospective cohort study evaluated patients with generalized anti-AChR myasthenia gravis (MG) failing current therapy enrolled in the French early access program (EAP) for zilucoplan. Patients were evaluated at enrollment and at Months 1, 3 and 6 with the MG-ADL score, the Myasthenic Muscle Score (MMS-Garches), and the MG-quality of life questionnaire (MG-QoL15r). MG crises and on-treatment adverse events were documented. MG medication use was compared between the six months before and after enrollment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Forty-eight patients were enrolled and treated with zilucoplan (mean age: 56.2 years; 50.9% men). Thirty patients achieved six months follow-up before the EAP ended. Three patients discontinued zilucoplan due to lack of efficacy. Mean MG-ADL score decreased from 7.4\u2009\u00b1\u20094.4 to M0 to 2.5\u2009\u00b1\u20092.7 at M6 (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009&lt;\u20090.0001). 9\/43 patients achieved minimal symptom expression at M3 and 12\/30 at M6. Mean MMS-Garches score was 67.5\u2009\u00b1\u200920.0 at M0 and 87.5\u2009\u00b1\u200913.2 at M6 (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009&lt;\u20090.0001). Mean MG-QoL15r score was 18.2\u2009\u00b1\u20098.6 at M0 and 8.7\u2009\u00b1\u20098.3 at M6 (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009=\u20090.0156). 15 patients experienced adverse events, leading to discontinuation in four cases. The mean oral corticosteroid dose decreased from 28.8\u2009\u00b1\u200911.8\u2005mg to 17.9\u2009\u00b1\u200911.8 (\n                      <jats:italic toggle=\"yes\">p<\/jats:italic>\n                      \u2009\n                      <jats:italic toggle=\"yes\">=<\/jats:italic>\n                      \u20090.001). Six patients experienced MG crises on treatment, compared to 22 in the six months preceding enrollment. treatment.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Following initiation of zilucoplan treatment, we observed an improvement in MG symptoms and a reduction in MG crises and in exposure to oral corticosteroids. Overall, zilucoplan was well-tolerated with no major adverse events reported.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial registration information<\/jats:title>\n                    <jats:p>\n                      The study was registered in the clinicaltrials.gov trial registry under the number NCT06815133 (\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT06815133\">https:\/\/clinicaltrials.gov\/study\/NCT06815133<\/jats:ext-link>\n                      ) on February 4\n                      <jats:sup>th<\/jats:sup>\n                      , 2025.\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/22143602261437792","type":"journal-article","created":{"date-parts":[[2026,3,29]],"date-time":"2026-03-29T22:01:33Z","timestamp":1774821693000},"page":"1313-1325","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":3,"title":["Real-world effectiveness and safety of zilucoplan in patients with anti-AChR myasthenia gravis: A retrospective cohort study in France"],"prefix":"10.1177","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0009-0007-4759-5266","authenticated-orcid":false,"given":"Giulia","family":"Tammam","sequence":"first","affiliation":[{"name":"Nice University Hospital, Pasteur 2 Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0008-8418-0307","authenticated-orcid":false,"given":"Abderhmane","family":"Slioui","sequence":"additional","affiliation":[{"name":"Nice University Hospital, Pasteur 2 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Hospital"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Guilhem","family":"Sole","sequence":"additional","affiliation":[{"name":"Pellegrin Hospital, Bordeaux University Hospitals"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Baptiste","family":"Noury","sequence":"additional","affiliation":[{"name":"University of Western Brittany"},{"name":"Reference Center for Neuromuscular Disorders, FILNEMUS, Brest Regional University Hospital, BREST, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luce","family":"Barbat Du Closel","sequence":"additional","affiliation":[{"name":"La Timone Hospital University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3266-0623","authenticated-orcid":false,"given":"Cl\u00e9mence","family":"Marois","sequence":"additional","affiliation":[{"name":"Sorbonne Universit\u00e9"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aleksandra 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