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New SMA therapies could mark a paradigm shift in scoliosis management, but their effects on scoliosis development remain unclear. This study aims to observe scoliosis progression in the current treatment landscape to inform management strategies.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Methods<\/jats:title>\n            <jats:p>We conducted a cross-sectional retrospective analysis of 94 SMA patients treated at our center. Scoliosis development was evaluated in 75 patients using spine radiographs and electronic health records. Statistical analysis was performed using Python and GraphPad Prism. One-way ANOVA and Pearson correlation were used for group comparisons and correlation analysis, respectively.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Results<\/jats:title>\n            <jats:p>Scoliosis parameters in 5q-SMA patients who had received either nusinersen, onasemnogene abeparvovec, risdiplam, or their combinations showed mean ages at scoliosis detection were 23.94, 55.52, and 168.11\u00a0months for SMA types 1, 2, and 3, respectively. Cobb angles at detection showed no significant intergroup differences. The mean ages at scoliosis surgery were 60, 88.43, and 124.8\u00a0months. Pelvic obliquity (PO) was highest in type 1 and lowest in type 3. A strong correlation (r\u2009=\u20090.9) was found between PO measurement techniques. HFMSE scores correlated moderately with scoliosis severity (r\u2009=\u2009-0.38), while CHOP-INTEND showed no correlation.<\/jats:p>\n          <\/jats:sec>\n          <jats:sec>\n            <jats:title>Conclusion<\/jats:title>\n            <jats:p>The observations made in this study suggest that the effects of SMA therapies do not prevent scoliosis development. The improved prognosis may lead to a growing cohort of SMA type 1 and 2 patients with early onset scoliosis who require early growth-friendly surgical interventions.<\/jats:p>\n          <\/jats:sec>","DOI":"10.1007\/s00586-024-08586-y","type":"journal-article","created":{"date-parts":[[2024,12,31]],"date-time":"2024-12-31T11:49:34Z","timestamp":1735645774000},"page":"546-555","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Scoliosis development in 5q-spinal muscular atrophy under disease modifying therapies"],"prefix":"10.1007","volume":"34","author":[{"given":"Tu-Lan","family":"Vu-Han","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Wei\u00df","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"K\u00f6hli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lukas","family":"Sch\u00f6nnagel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carsten","family":"Perka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthias","family":"Pumberger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,12,31]]},"reference":[{"key":"8586_CR1","doi-asserted-by":"publisher","first-page":"1713","DOI":"10.1056\/NEJMoa1706198","volume":"377","author":"JR Mendell","year":"2017","unstructured":"Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L\u2019Italien J, Sproule DM, Wells C, Cardenas JA, Heitzer MD, Kaspar A, Corcoran S, Braun L, Likhite S, Miranda C, Meyer K, Foust KD, Burghes AHM, Kaspar BK (2017) Single-dose gene-replacement therapy for spinal muscular atrophy. 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TV receives funding for her research time by the BIH Charit\u00e9 Digital Clinician Scientist Program. The remaining authors do not have any conflicts of interests related to this article to declare.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}},{"value":"This study has been approved by Charit\u00e9 Universit\u00e4tsmedizin Berlin Ethics Commission (EA2\/178\/21) and has been performed in accordance with the ethical standards laid down in the 1964 Declaration of Helsinki and its later amendments. Clinical patient data collected within this study has been collected with informed consent from all subjects and\/or their legal guardian(s).","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical approval"}}]}}