{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T11:17:01Z","timestamp":1790767021301,"version":"4.1.0"},"reference-count":22,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2022,11,12]],"date-time":"2022-11-12T00:00:00Z","timestamp":1668211200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100003157","name":"Myasthenia Gravis Foundation of America","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100003157","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2023,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Within the last 5\u2009years, the US Food and Drug Administration (FDA) has approved complement and neonatal Fc receptor (FcRN) inhibitors for treatment of generalized myasthenia gravis, and several other therapies are in late\u2010stage clinical trials or under regulatory review. However, questions about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, has resulted in uncertainty around the place that they should occupy in the existing therapeutic armamentarium. MGNet (a Rare Diseases Clinical Research Consortium funded by the National Institute of Neurological Diseases and Stroke) held two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for myasthenia gravis. Herein we provide a summary of these discussions, but not a consensus opinion, and offer a series of recommendations to guide focused research in the most critical areas. We welcome ongoing discussion through comments on this work.<\/jats:p>","DOI":"10.1002\/mus.27742","type":"journal-article","created":{"date-parts":[[2022,11,2]],"date-time":"2022-11-02T12:12:29Z","timestamp":1667391149000},"page":"12-16","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["The best and worst of times in therapy development for myasthenia gravis"],"prefix":"10.1002","volume":"67","author":[{"given":"Michael","family":"Benatar","sequence":"first","affiliation":[{"name":"Department of Neurology University of Miami  Miami Florida"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gary","family":"Cutter","sequence":"additional","affiliation":[{"name":"Department of Biostatistics University of Alabama at Birmingham  Birmingham Alabama"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8195-0141","authenticated-orcid":false,"given":"Henry J.","family":"Kaminski","sequence":"additional","affiliation":[{"name":"Department of Neurology &amp; Rehabilitation Medicine George Washington University  Washington DC"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,11,12]]},"reference":[{"key":"e_1_2_14_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(17)30369-1"},{"key":"e_1_2_14_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00159-9"},{"key":"e_1_2_14_4_1","doi-asserted-by":"publisher","DOI":"10.1001\/jamaneurol.2019.5125"},{"key":"e_1_2_14_5_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-022-01726-y"},{"key":"e_1_2_14_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00463-4"},{"key":"e_1_2_14_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000011124"},{"key":"e_1_2_14_8_1","doi-asserted-by":"publisher","DOI":"10.12659\/MSM.933296"},{"key":"e_1_2_14_9_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2022.886625"},{"key":"e_1_2_14_10_1","first-page":"119","article-title":"The effectiveness and value of eculizumab and efgartigimod for generalized myasthenia gravis","volume":"28","author":"Tice JA","year":"2022","journal-title":"J Manag Care Spec Pharm."},{"key":"e_1_2_14_11_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620719874728"},{"key":"e_1_2_14_12_1","unstructured":"KansteinerF.Argenx already deep into its Vyvgart launch charts course to become immunology 'powerhouse': CEO.2022.https:\/\/www.fiercepharma.com\/pharma\/argenx-fresh-off-fda-nod-for-vyvgart-charts-course-to-become-immunology-powerhouse-ceo. 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Accessed September 26 2022."},{"key":"e_1_2_14_20_1","doi-asserted-by":"publisher","DOI":"10.1177\/1352458519848965"},{"key":"e_1_2_14_21_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27454"},{"key":"e_1_2_14_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.23330"},{"key":"e_1_2_14_23_1","doi-asserted-by":"publisher","DOI":"10.1093\/ije\/dyu045"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27742","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27742","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27742","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,18]],"date-time":"2023-08-18T21:03:28Z","timestamp":1692392608000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27742"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,11,12]]},"references-count":22,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2023,1]]}},"alternative-id":["10.1002\/mus.27742"],"URL":"https:\/\/doi.org\/10.1002\/mus.27742","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2022,11,12]]},"assertion":[{"value":"2022-08-02","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-10-25","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2022-11-12","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}