{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,1]],"date-time":"2026-11-01T03:07:39Z","timestamp":1793502459000,"version":"4.5.0"},"reference-count":30,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T00:00:00Z","timestamp":1694131200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T00:00:00Z","timestamp":1694131200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100004913","name":"Universit\u00e0 degli Studi di Palermo","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100004913","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Stiff-person syndrome (SPS) is a rare autoimmune neurological disorder characterized by high titers of antibodies against glutamic acid decarboxylase (GAD) causing impaired GABAergic inhibitory neurotransmission. To date, there is not a defined therapy for such condition, but immunomodulating therapies, such as plasma exchange, intravenous immunoglobulins, and rituximab, have been widely used in clinical practice. However, the efficacy and tolerability of these treatments is not well established. Efgartigimod, a new neonatal Fc receptor (FcRn) blocker, is a human IgG1 antibody Fc fragment engineered with increased affinity for FcRn binding, leading\u00a0to a\u00a0reduction in IgGs levels, including\u00a0pathogenic IgG autoantibody showing promising results in neurological autoimmune disorders and has been approved for the treatment of AChR-seropositive generalized\u00a0myasthenia gravis (MG). In this study, we report and describe the first data on treatment with efgartigimod in three patients affected by both AChR-seropositive generalized MG and anti-GAD-seropositive SPS. Patients were followed since the start of efgartigimod and for the whole treatment period (12\u00a0weeks). MG symptoms were assessed with the \u201cMG activity of daily living score\u201d and the Quantitative Myasthenia Gravis score, while SPS ones were assessed with the \u201cSPS activity of daily living score\u201d; muscle strength was assessed with the Medical Research Council Sum score; the overall disability from MG and SPS was assessed by the modified Rankin Scale. All patients showed an improvement in symptoms of both SPS and MG after 2 cycles of treatment. Our data suggest that efgartigimod may be considered as a candidate drug for SPS and other autoantibody-mediated neurological disorders.<\/jats:p>","DOI":"10.1007\/s00415-023-11970-1","type":"journal-article","created":{"date-parts":[[2023,9,8]],"date-time":"2023-09-08T13:03:06Z","timestamp":1694178186000},"page":"254-262","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":55,"title":["Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome"],"prefix":"10.1007","volume":"271","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9805-1655","authenticated-orcid":false,"given":"Vincenzo","family":"Di Stefano","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo","family":"Alonge","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicasio","family":"Rini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Massimiliano","family":"Militello","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonino","family":"Lupica","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angelo","family":"Torrente","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Filippo","family":"Brighina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2023,9,8]]},"reference":[{"key":"11970_CR1","doi-asserted-by":"crossref","unstructured":"Dalakas MC (2022) Stiff-person Syndrome and GAD Antibody-spectrum Disorders: GABAergic Neuronal Excitability, Immunopathogenesis and Update on Antibody Therapies. Neurotherapeutics [Internet]. 2022 Apr 1 [cited 2023 May 4];19(3):832\u201347. Available from: https:\/\/pubmed.ncbi.nlm.nih.gov\/35084720\/","DOI":"10.1007\/s13311-022-01188-w"},{"key":"11970_CR2","doi-asserted-by":"crossref","unstructured":"Tsiortou P, Alexopoulos H, Dalakas MC (2021) GAD antibody-spectrum disorders: progress in clinical phenotypes, immunopathogenesis and therapeutic interventions. Ther Adv Neurol Disord [Internet]. 2021 [cited 2023 May 4];14. Available from: https:\/\/pubmed.ncbi.nlm.nih.gov\/33854562\/","DOI":"10.1177\/17562864211003486"},{"key":"11970_CR3","doi-asserted-by":"crossref","unstructured":"Dade M, Berzero G, Izquierdo C, Giry M, Benazra M, Delattre JY et al (2020) Neurological syndromes associated with anti-GAD antibodies. Int J Mol Sci [Internet]. 2020 May 2 [cited 2023 May 4];21(10). Available from: https:\/\/pubmed.ncbi.nlm.nih.gov\/32456344\/","DOI":"10.3390\/ijms21103701"},{"key":"#cr-split#-11970_CR4.1","doi-asserted-by":"crossref","unstructured":"Dalakas MC (2023) Therapies in Stiff-Person Syndrome: Advances and Future Prospects Based on Disease Pathophysiology. Neurology","DOI":"10.1212\/NXI.0000000000200109"},{"key":"#cr-split#-11970_CR4.2","unstructured":"(R) neuroimmunology & neuroinflammation [Internet]. 2023 May 1 [cited 2023 May 4];10(3). Available from: https:\/\/pubmed.ncbi.nlm.nih.gov\/37059468\/"},{"key":"11970_CR5","doi-asserted-by":"publisher","unstructured":"Di Stefano V, Barbone F, Ferrante C, Telese R, Vitale M, Onofrj M et al (2020) Inflammatory polyradiculoneuropathies: Clinical and immunological aspects, current therapies, and future perspectives. Eur J Inflamm [Internet]. 2020 Jan 27 [cited 2021 Mar 7];18:205873922094234. 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