{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T03:53:42Z","timestamp":1777953222324,"version":"3.51.4"},"reference-count":22,"publisher":"Wiley","issue":"6","license":[{"start":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T00:00:00Z","timestamp":1772150400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T00:00:00Z","timestamp":1772150400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2026,6]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction\/Aims<\/jats:title>\n                    <jats:p>\n                      Data comparing nusinersen monotherapy with combination therapy using nusinersen and onasemnogene abeparvovec (OA) in spinal muscular atrophy (SMA) type 1 patients are limited. This study aimed to compare the clinical outcomes of nusinersen monotherapy versus combination therapy (nusinersen and OA) in SMA type 1 patients with two\n                      <jats:italic>SMN2<\/jats:italic>\n                      copies.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      This retrospective multicenter study included 82 patients divided into nusinersen monotherapy (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200942) and combination therapy (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200940) groups. Outcomes included motor milestones, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP\u2010INTEND) scores, respiratory and nutritional status, and survival. Patients were stratified by age at treatment initiation (\u2264\u20093, 3\u20136, and &gt;\u20096\u2009months). An age\u2010matched subgroup analysis was performed.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Age at treatment initiation, total administered nusinersen doses, and follow\u2010up duration were comparable between groups. All deaths (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u20098) occurred in the monotherapy group among patients who initiated treatment after 3\u2009months of age (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.005). There was no significant difference between groups in the age at achieving unsupported sitting. In the age\u2010matched subgroup analysis (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200944, 22 per group), no significant differences were found between monotherapy and combination therapy in terms of motor function, CHOP\u2010INTEND progression, respiratory and feeding status. Earlier treatment was associated with better feeding outcomes (OR: 0.740,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.024). Pre\u2010treatment respiratory and feeding status were also predictive of corresponding outcomes.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Early treatment is the main determinant of outcomes, while combination therapy shows no clear advantage over monotherapy. Larger prospective studies are needed to clarify the role and timing of combination therapy.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/mus.70191","type":"journal-article","created":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T21:31:40Z","timestamp":1773005500000},"page":"966-975","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Comparison of Nusinersen Monotherapy Versus Combination Therapy With Nusinersen and Onasemnogene Abeparvovec in Spinal Muscular Atrophy Type 1 Patients With Two\n                    <i>SMN2<\/i>\n                    Copies: A Multicenter Study From T\u00fcrkiye"],"prefix":"10.1002","volume":"73","author":[{"given":"Hulya Maras","family":"Genc","sequence":"first","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics, Istanbul Faculty of Medicine Istanbul University  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Fulya","family":"Kurekci","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Sanliurfa Training and Research Hospital  Sanliurfa T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gulten","family":"Ozturk","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Marmara University School of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1821-6881","authenticated-orcid":false,"given":"Ayfer Sakarya","family":"Gunes","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Kocaeli University Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sema","family":"Saltik","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Istanbul University\u2010Cerrahpa\u015fa Cerrahpa\u015fa Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6571-8449","authenticated-orcid":false,"given":"Mehmet Akif","family":"Kilic","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics, Istanbul Faculty of Medicine Istanbul University  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gokce","family":"Cirdi","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Kocaeli University Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Olcay","family":"Unver","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Marmara University School of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mustafa","family":"Komur","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Mersin University Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ismail Hakki","family":"Akbeyaz","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Marmara University School of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Y\u0131lmaz","family":"Zindar","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Istanbul University\u2010Cerrahpa\u015fa Cerrahpa\u015fa Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leman Tekin","family":"Orgun","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Kocaeli University Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cisem Duman","family":"Kayar","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics, Istanbul Faculty of Medicine Istanbul University  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Serhat","family":"Guler","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Istanbul University\u2010Cerrahpa\u015fa Cerrahpa\u015fa Faculty of Medicine  Istanbul 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Neurology, Department of Pediatrics Kocaeli University Faculty of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Dilsad","family":"Turkdogan","sequence":"additional","affiliation":[{"name":"Division of Pediatric Neurology, Department of Pediatrics Marmara University School of Medicine  Istanbul T\u00fcrkiye"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"name":"Marmara Region SMA Study 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