{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,6]],"date-time":"2026-11-06T00:28:18Z","timestamp":1793924898217,"version":"4.8.0"},"reference-count":37,"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><jats:title>Background:<\/jats:title><jats:p> Muscle-specific tyrosine kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) is a rare and frequently severe subtype of gMG. <\/jats:p><\/jats:sec><jats:sec><jats:title>Objectives:<\/jats:title><jats:p> To assess the efficacy and safety of rozanolixizumab in the subgroup of patients with MuSK Ab+ gMG in the MycarinG study. <\/jats:p><\/jats:sec><jats:sec><jats:title>Design:<\/jats:title><jats:p> A randomised, double-blind, placebo-controlled phase III study. <\/jats:p><\/jats:sec><jats:sec><jats:title>Methods:<\/jats:title><jats:p> Patients with acetylcholine receptor (AChR) Ab+ or MuSK Ab+ gMG (aged \u2a7e18 years, Myasthenia Gravis Foundation of America Disease Class II\u2013IVa, Myasthenia Gravis Activities of Daily Living [MG-\u200dADL] score \u2a7e3.0 [non-ocular symptoms], Quantitative Myasthenia Gravis score \u2a7e11.0) were randomly assigned (1:1:1) to receive once-weekly subcutaneous infusions of rozanolixizumab 7\u2009mg\/kg, rozanolixizumab 10\u2009mg\/kg or placebo for 6\u2009weeks, followed by an 8-week observation period. Randomisation was stratified by AChR and MuSK autoantibody status. The primary study endpoint was change from baseline to Day 43 in MG-ADL score. Treatment-emergent adverse events (TEAEs) were also assessed. <\/jats:p><\/jats:sec><jats:sec><jats:title>Results:<\/jats:title><jats:p> Overall, 200 patients were randomised, of whom 21 had MuSK Ab+ gMG and received rozanolixizumab 7\u2009mg\/kg ( n\u2009=\u20095), 10\u2009mg\/kg ( n\u2009=\u20098) or placebo ( n\u2009=\u20098). In patients with MuSK Ab+ gMG, reductions from baseline to Day 43 in MG-ADL scores were observed: rozanolixizumab 7\u2009mg\/kg least squares mean (LSM) change (standard error), \u20137.28 (1.94); 10\u2009mg\/kg, \u20134.16 (1.78); and placebo, 2.28 (1.95). Rozanolixizumab 7\u2009mg\/kg LSM difference from placebo was \u22129.56 (97.5% confidence interval: \u221215.25, \u22123.87); 10\u2009mg\/kg, \u22126.45 (\u221211.03, \u20131.86). TEAEs were experienced by four (80.0%), five (62.5%) and three (37.5%) patients with MuSK Ab+ gMG receiving rozanolixizumab 7\u2009mg\/kg, 10\u2009mg\/kg and placebo, respectively. No patients experienced serious TEAEs. No deaths occurred. <\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion:<\/jats:title><jats:p> This subgroup analysis of adult patients with MuSK Ab+ gMG enrolled in the MycarinG study supports the use of rozanolixizumab as an effective treatment option for patients with gMG who have MuSK autoantibodies. <\/jats:p><\/jats:sec><jats:sec><jats:title>Trial registration:<\/jats:title><jats:p> ClinicalTrials.gov: NCT03971422 ( https:\/\/clinicaltrials.gov\/study\/NCT03971422 ); EU Clinical Trials Register: EudraCT 2019-000968-18 ( https:\/\/www.clinicaltrials\u200cregister.eu\/ctr-search\/trial\/2019-000968-18\/GB ). <\/jats:p><\/jats:sec>","DOI":"10.1177\/17562864241273036","type":"journal-article","created":{"date-parts":[[2024,9,14]],"date-time":"2024-09-14T02:42:24Z","timestamp":1726281744000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":17,"title":["Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3432-9747","authenticated-orcid":false,"given":"Ali A.","family":"Habib","sequence":"first","affiliation":[{"name":"MDA ALS and Neuromuscular Center, University of California, 200 South Manchester Avenue, Suite 110, Irvine, Orange, CA 92868, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sabrina","family":"Sacconi","sequence":"additional","affiliation":[{"name":"Peripheral Nervous System & Muscle Department, Pasteur 2 Hospital, Centre Hospitalier Universitaire de Nice, Universit\u00e9 C\u00f4te d\u2019Azur, Nice, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanni","family":"Antonini","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Mental Health and Sensory Organs (NESMOS), Sapienza University of Rome, Rome, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Cort\u00e9s-Vicente","sequence":"additional","affiliation":[{"name":"Neuromuscular Diseases Unit, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julian","family":"Grosskreutz","sequence":"additional","affiliation":[{"name":"Precision Neurology of Neuromuscular Diseases, Department of Neurology, University of L\u00fcbeck, L\u00fcbeck, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0002-4864-8044","authenticated-orcid":false,"given":"Zabeen K.","family":"Mahuwala","sequence":"additional","affiliation":[{"name":"Department of Neuromuscular Medicine, Epilepsy and Clinical Neurophysiology, University of Kentucky, Lexington, KY, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9810-5737","authenticated-orcid":false,"given":"Renato","family":"Mantegazza","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione IRCCS, Istituto Nazionale Neurologico Carlo Besta, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert M.","family":"Pascuzzi","sequence":"additional","affiliation":[{"name":"Department of Neurology, Indiana University School of Medicine, Indiana University Health, Indianapolis, IN, USA"}],"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":"John","family":"Vissing","sequence":"additional","affiliation":[{"name":"Department of Neurology, 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