{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,21]],"date-time":"2026-10-21T17:30:52Z","timestamp":1792603852963,"version":"4.5.0"},"reference-count":60,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2022,12,13]],"date-time":"2022-12-13T00:00:00Z","timestamp":1670889600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100008530","name":"European Regional Development Fund","doi-asserted-by":"publisher","award":["KK01.1.1.01.0007"],"award-info":[{"award-number":["KK01.1.1.01.0007"]}],"id":[{"id":"10.13039\/501100008530","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100004488","name":"Hrvatska Zaklada za Znanost","doi-asserted-by":"publisher","award":["2019\u201004\u20103584"],"award-info":[{"award-number":["2019\u201004\u20103584"]}],"id":[{"id":"10.13039\/501100004488","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["CNS Neurosci Ther"],"published-print":{"date-parts":[[2024,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Aims<\/jats:title><jats:p>Considering the substantial variability in treatment response across patients with spinal muscular atrophy (SMA), reliable markers for monitoring response to therapy and predicting treatment responders need to be identified. The study aimed to determine if measured concentrations of disease biomarkers (total tau protein, neurofilament light chain, and S100B protein) correlate with the duration of nusinersen treatment and with scores obtained using functional scales for the assessment of motor abilities.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>A total of 30 subjects with SMA treated with nusinersen between 2017 and 2021 at the Department of Pediatrics, University Hospital Centre Zagreb, Croatia, were included in this study. Cerebrospinal fluid (CSF) samples were collected by lumbar puncture prior to intrathecal application of nusinersen. Protein concentrations in CSF samples were determined by enzyme\u2010linked immunosorbent assay in 26 subjects. The motor functions were assessed using functional motor scales.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The main finding was significantly decreased total tau correlating with the number of nusinersen doses and motor improvement in the first 18\u201324\u2009months of treatment (in all SMA patients and SMA type 1 patients). Neurofilament light chain and S100B were not significantly changed after administration of nusinersen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>The measurement of total tau concentration in CSF is a reliable index for monitoring the biomarker and clinical response to nusinersen therapy in patients with SMA.<\/jats:p><\/jats:sec>","DOI":"10.1111\/cns.14051","type":"journal-article","created":{"date-parts":[[2022,12,14]],"date-time":"2022-12-14T04:16:35Z","timestamp":1670991395000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Total tau in cerebrospinal fluid detects treatment responders among spinal muscular atrophy types 1\u20133 patients treated with nusinersen"],"prefix":"10.1111","volume":"30","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6339-9261","authenticated-orcid":false,"given":"Goran","family":"\u0160imi\u0107","sequence":"first","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vana","family":"Vuki\u0107","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marija","family":"Babi\u0107","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Banovi\u0107","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivana","family":"Bere\u010di\u0107","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ena","family":"\u0160pani\u0107","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Klara","family":"Zub\u010di\u0107","sequence":"additional","affiliation":[{"name":"Department of Neuroscience, Croatian Institute for Brain Research University of Zagreb School of Medicine  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anja Tea","family":"Golubi\u0107","sequence":"additional","affiliation":[{"name":"Department of Nuclear Medicine and Radiation Protection University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marija","family":"Bari\u0161i\u0107 Kutija","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Merkler \u0160orgi\u0107","sequence":"additional","affiliation":[{"name":"Department of Laboratory Diagnostics, Laboratory for Molecular Diagnostics University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"\u017deljka","family":"Vogrinc","sequence":"additional","affiliation":[{"name":"Department of Laboratory Diagnostics University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivan","family":"Lehman","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University Hospital Centre Zagreb  Zagreb Croatia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3208-1154","authenticated-orcid":false,"given":"Patrick R.","family":"Hof","sequence":"additional","affiliation":[{"name":"Nash Family Department of Neuroscience, Friedman Brain Institute, and Ronald M. 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