{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,29]],"date-time":"2026-10-29T23:31:11Z","timestamp":1793316671305,"version":"4.5.0"},"reference-count":26,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2018,1,1]],"date-time":"2018-01-01T00:00:00Z","timestamp":1514764800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"name":"Catalyst Pharmaceuticals"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["SAGE Open Medicine"],"published-print":{"date-parts":[[2018,1]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>The aim of this study is to determine the safety and the efficacy of amifampridine phosphate in muscle-specific kinase antibody-positive myasthenia gravis, in a 1:1 randomized, double-blind, placebo-controlled, switchback, double crossover study.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Eligible patients had muscle-specific kinase myasthenia gravis, &gt;18 years of age, and Myasthenia Gravis Foundation of America class II\u2013IV with a score of \u2a7e9 on Myasthenia Gravis Composite scale. After the run-in phase, during which amifampridine phosphate was titrated to a tolerable and effective dosage, patients were randomized to receive placebo\u2013amifampridine\u2013placebo sequence or amifampridine\u2013placebo\u2013amifampridine sequence daily for 7\u2009days. Then, patients switched treatment arms twice, for a total of 21\u2009days of double-blind treatment. Safety was determined by serial assessments of adverse events\/serious adverse events, physical examinations, and clinical and laboratory tests. The co-primary outcome measures included changes from baseline of Quantitative Myasthenia Gravis score and Myasthenia Gravis\u2013specific Activities of Daily Living Profile score. The secondary outcome measures comprised changes from baseline of Myasthenia Gravis Composite score, Myasthenia Gravis Quality of Life scale\u201415 questions, Fatigue Severity Scale, and Carlo Besta Neurological Institute\u2013Myasthenia Gravis scale. Statistical analyses were assessed using a switchback model for three-period, two-treatment crossover design.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>A total of 10 patients were screened, enrolled, and treated. Transient paresthesias (60%) were the only amifampridine phosphate\u2013related adverse events reported. Four patients were randomized to receive placebo\u2013amifampridine\u2013placebo sequence and three patients to receive amifampridine\u2013placebo\u2013amifampridine sequence. The co-primary objectives were statistically met (Quantitative Myasthenia Gravis score: p\u2009=\u20090.0003 and Myasthenia Gravis\u2013specific Activities of Daily Living Profile score: p\u2009=\u20090.0006), as well as all the secondary endpoints (Myasthenia Gravis Composite score: p\u2009&lt;\u20090.0001, Myasthenia Gravis Quality of Life scale\u201415 questions: p\u2009=\u20090.0025, Fatigue Severity Scale: p\u2009=\u20090.0061, and Carlo Besta Neurological Institute\u2013Myasthenia Gravis scale: p\u2009=\u20090.0014).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>Despite the low number of patients, MuSK-001 study provided evidence that amifampridine phosphate, in the range of 30\u201360\u2009mg daily dose, was safe and effective in treating muscle-specific kinase myasthenia gravis, suggesting the need for a large multi-center trial to confirm these results.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/2050312118819013","type":"journal-article","created":{"date-parts":[[2018,12,17]],"date-time":"2018-12-17T08:05:48Z","timestamp":1545033948000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":28,"title":["Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study"],"prefix":"10.1177","volume":"6","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8823-6821","authenticated-orcid":false,"given":"Silvia","family":"Bonanno","sequence":"first","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria Barbara","family":"Pasanisi","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rita","family":"Frangiamore","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo","family":"Maggi","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlo","family":"Antozzi","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Francesca","family":"Andreetta","sequence":"additional","affiliation":[{"name":"Department of Neuroimmunology and Neuromuscular Diseases, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Campanella","sequence":"additional","affiliation":[{"name":"Department of Clinical Research and Innovation, Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Greta","family":"Brenna","sequence":"additional","affiliation":[{"name":"SPARC Consulting Srl, Milan, Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo","family":"Cottini","sequence":"additional","affiliation":[{"name":"High Research Srl, Milan, Italy"}],"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 I.R.C.C.S. Istituto Neurologico Carlo Besta (INCB), Milan, Italy"},{"name":"Department of Clinical Research and Innovation, Fondazione I.R.C.C.S. 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