{
  "abstract": "Importance: Gefurulimab is a novel dual-binding nanobody that blocks complement component 5 activation. Complement activation is a key pathogenic mechanism in anti-acetylcholine receptor antibody-positive (AChR-Ab+) generalized myasthenia gravis (gMG). Objective: To evaluate the efficacy and safety of gefurulimab in adults with AChR-Ab+ gMG. Design, Setting, and Participants: 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 ≥18 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. Intervention: Gefurulimab or placebo via once-weekly subcutaneous self-injection. Main Outcomes and Measures: 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. Results: Of 405 patients screened, 145 were excluded; 260 met eligibility criteria and were randomized (gefurulimab, n = 131; placebo, n = 129); 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 -4.2 vs -2.6 (treatment difference, -1.6; 95% CI, -2.4 to -0.8; P < .001) and -4.5 vs -2.4 (treatment difference, -2.1; 95% CI, -3.1 to -1.1; P < .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. Conclusions and Relevance: 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. Trial Registration: ClinicalTrials.gov Identifier: NCT05556096.",
  "abstract_shown": true,
  "affiliations": [],
  "article_type": null,
  "arxiv": null,
  "author_total": 126,
  "authors": [
    {
      "name": "Kelly G. Gwathmey"
    },
    {
      "name": "Francesco Saccà"
    },
    {
      "name": "James F. Howard"
    },
    {
      "name": "Tuan Vu"
    },
    {
      "name": "Jianying Xi"
    },
    {
      "name": "Mathias Mäurer"
    },
    {
      "name": "Tobias Ruck"
    },
    {
      "name": "Ha Young Shin"
    },
    {
      "name": "Masanori P. Takahashi"
    },
    {
      "name": "Carlos Casasnovas"
    },
    {
      "name": "Marek Śmiłowski"
    },
    {
      "name": "Stojan Peric"
    },
    {
      "name": "Masayuki Masuda"
    },
    {
      "name": "Joachim Scholz"
    },
    {
      "name": "Shulian Shang"
    },
    {
      "name": "Min Yee"
    },
    {
      "name": "Sanjay Rakhade"
    },
    {
      "name": "Djillali Annane"
    },
    {
      "name": "Prevail Trial Investigators"
    },
    {
      "name": "Wolfgang Löscher"
    },
    {
      "name": "Carolina Mainella"
    },
    {
      "name": "Raffi Topakian"
    },
    {
      "name": "Wilson Marques"
    },
    {
      "name": "Maria de Penha Morita"
    },
    {
      "name": "Edmar Zanoteli"
    },
    {
      "name": "Marcela Costa"
    },
    {
      "name": "Roberto Rey"
    },
    {
      "name": "Alexandre Guerreiro"
    },
    {
      "name": "Alexandre Longo"
    },
    {
      "name": "Raffaele Iorio"
    },
    {
      "name": "Carolina Barnett-Tapia"
    },
    {
      "name": "Vera Bril"
    },
    {
      "name": "Zaeem Siddiqi"
    },
    {
      "name": "Annie Dionne"
    },
    {
      "name": "Matteo Garibaldi"
    },
    {
      "name": "Marcelo Rugiero"
    },
    {
      "name": "Hui Deng"
    },
    {
      "name": "Hongyu Zhou"
    },
    {
      "name": "Ruisheng Duan"
    },
    {
      "name": "Nicolai Rasmus Preisler"
    },
    {
      "name": "Qun Xue"
    },
    {
      "name": "Huan Yang"
    },
    {
      "name": "Zhangyu Zhou"
    },
    {
      "name": "Ting Chang"
    },
    {
      "name": "Bitao Bu"
    },
    {
      "name": "Qing Ke"
    },
    {
      "name": "Akira Tsujino"
    },
    {
      "name": "Akiyuki Uzawa"
    },
    {
      "name": "Hua Zhang"
    },
    {
      "name": "Chuanzhu Yan"
    },
    {
      "name": "Jin Wang"
    },
    {
      "name": "Feng Gao"
    },
    {
      "name": "Céline Tard"
    },
    {
      "name": "Florencia Aguirre"
    },
    {
      "name": "Shahram Attarian"
    },
    {
      "name": "Michelangelo Maestri"
    },
    {
      "name": "Saskia Bresch"
    },
    {
      "name": "Valentina Damato"
    },
    {
      "name": "Shahar Shelly"
    },
    {
      "name": "Aleksandra Nadaj-Pakleza"
    }
  ],
  "bibtex": "https://research.draconitic.com/paper/f1tegbrnv8zwm.bib",
  "cited_by": 5,
  "doi": "10.1001/jamaneurol.2026.2333",
  "fulltext_url": null,
  "id": "f1tegbrnv8zwm",
  "issns": [
    "2168-6149",
    "2168-6157"
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  "landing_url": "https://hal.science/hal-05850310v1",
  "language": "en",
  "licence": "CC-BY-NC-ND",
  "licence_label": "CC BY-NC-ND",
  "licence_source": "journal",
  "licence_url": "https://creativecommons.org/licenses/by-nc-nd/4.0/",
  "metadata_licence": null,
  "native_subjects": [
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      "openalex",
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  "openalex": "W7171397801",
  "pdf_url": null,
  "pmcid": null,
  "pmid": "42507440",
  "published": "2026-11-03",
  "publisher": "American Medical Association",
  "ris": "https://research.draconitic.com/paper/f1tegbrnv8zwm.ris",
  "sources": [
    {
      "first_seen": "2026-11-04T12:20:52Z",
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  "subjects": [
    {
      "field": "medicine",
      "origin": "openalex:2728|Myasthenia Gravis and Thymoma",
      "subfield": "neurology"
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  "title": "Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis",
  "title_text": "Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis",
  "type": "peer-reviewed",
  "updated_at": "2026-11-05T12:20:12Z",
  "url": "https://research.draconitic.com/paper/f1tegbrnv8zwm",
  "venue": "JAMA neurology",
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}