{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T03:03:42Z","timestamp":1793070222324,"version":"4.5.0"},"reference-count":50,"publisher":"American Medical Association (AMA)","issue":"9","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JAMA Neurol"],"published-print":{"date-parts":[[2026,9,1]]},"abstract":"<jats:sec>\n                    <jats:title>Importance<\/jats:title>\n                    <jats:p>Gefurulimab is a novel dual-binding nanobody that blocks complement component 5 activation. Complement activation is a key pathogenic mechanism in anti\u2013acetylcholine receptor antibody\u2013positive (AChR-Ab+) generalized myasthenia gravis (gMG).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objective<\/jats:title>\n                    <jats:p>To evaluate the efficacy and safety of gefurulimab in adults with AChR-Ab+ gMG.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design, Setting, and Participants<\/jats:title>\n                    <jats:p>This randomized clinical trial, PREVAIL, was a phase 3, double-blind, placebo-controlled study conducted at 113 sites in 20 countries. Patients were screened and randomized from November 2022 to November 2024; the randomized controlled treatment period was 26 weeks. Participants were adults (aged \u226518 years) with AChR-Ab+ gMG, a Myasthenia Gravis Foundation of America classification II through IV, and a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score of 5 or higher.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Intervention<\/jats:title>\n                    <jats:p>Gefurulimab or placebo via once-weekly subcutaneous self-injection.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Main Outcomes and Measures<\/jats:title>\n                    <jats:p>The primary end point was change from baseline in MG-ADL total score at week 26. The key secondary end point was change from baseline in Quantitative Myasthenia Gravis (QMG) total score at week 26. Safety was also assessed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Of 405 patients screened, 145 were excluded; 260 met eligibility criteria and were randomized (gefurulimab, n\u2009=\u2009131; placebo, n\u2009=\u2009129); 249 patients completed the 26-week randomized controlled treatment period. The mean (SD) age was 52.8 (15.73) years; 157 (60.4%) patients were female and 103 (39.6%) were male. All primary and secondary end points were met with statistical significance. Improvements occurred within 1 week for MG-ADL score and 4 weeks for QMG score and were sustained through week 26. Least-squares mean change from baseline at week 26 for MG-ADL and QMG total scores for gefurulimab vs placebo were \u22124.2 vs \u22122.6 (treatment difference, \u22121.6; 95% CI, \u22122.4 to \u22120.8;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&amp;amp;lt;\u2009.001) and \u22124.5 vs \u22122.4 (treatment difference, \u22122.1; 95% CI, \u22123.1 to \u22121.1;\n                      <jats:italic>P<\/jats:italic>\n                      \u2009&amp;amp;lt;\u2009.001), respectively. The incidence of adverse events (AEs) was similar between groups. Most common treatment-emergent AEs with gefurulimab were injection site reactions, headache, back pain, and nasopharyngitis. No meningococcal infections were reported.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions and Relevance<\/jats:title>\n                    <jats:p>Gefurulimab demonstrated both early and sustained clinical benefit in AChR-Ab+ gMG and was well tolerated, supporting its potential as a convenient, once-weekly, self-administered treatment regimen.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trial Registration<\/jats:title>\n                    <jats:p>\n                      ClinicalTrials.gov Identifier:\n                      <jats:ext-link xmlns:xlink=\"http:\/\/www.w3.org\/1999\/xlink\" ext-link-type=\"uri\" xlink:href=\"https:\/\/clinicaltrials.gov\/study\/NCT05556096\">NCT05556096<\/jats:ext-link>\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1001\/jamaneurol.2026.2333","type":"journal-article","created":{"date-parts":[[2026,7,27]],"date-time":"2026-07-27T15:30:31Z","timestamp":1785166231000},"page":"841","source":"Crossref","is-referenced-by-count":5,"title":["Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis"],"prefix":"10.1001","volume":"83","author":[{"given":"Kelly G.","family":"Gwathmey","sequence":"first","affiliation":[{"name":"Virginia Commonwealth University, Richmond,"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesco","family":"Sacc\u00e0","sequence":"additional","affiliation":[{"name":"University of Naples Federico II, Napoli, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr","given":"James F.","family":"Howard","sequence":"additional","affiliation":[{"name":"University of North Carolina at Chapel Hill School of Medicine, Chapel 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