{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T01:48:14Z","timestamp":1790560094619,"version":"4.1.0"},"reference-count":30,"publisher":"Springer Science and Business Media LLC","issue":"11","license":[{"start":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T00:00:00Z","timestamp":1760918400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T00:00:00Z","timestamp":1760918400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/501100004375","name":"Tel Aviv University","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100004375","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Eur J Pediatr"],"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    Spinal muscular atrophy (SMA) is a progressive neuromuscular disorder characterized by respiratory decline. While nusinersen improves motor function, its long-term respiratory effects in milder SMA types (2 and 3) remain unclear. This study evaluates pulmonary outcomes following 3\u00a0years of treatment. We retrospectively reviewed genetically confirmed SMA 2 and 3 pediatric patients treated with nusinersen (2017\u20132022). Data included demographics, non-invasive ventilation (NIV) and mechanical insufflation-exsufflation (MIE) use, body mass index (BMI), feeding method, scoliosis, and respiratory hospitalizations. Pulmonary function tests (PFTs) at baseline and at year 3 included percent-predicted forced vital capacity (ppFVC), FEV1\/FVC ratio, peak cough flow (PCF), maximal voluntary ventilation (MVV), maximal inspiratory pressure (MIP), and maximal expiratory pressure (MEP). Included were 15 patients with type 2 and 13 patients with type 3 SMA. Their mean age at diagnosis was 3.5\u2009\u00b1\u20094\u00a0years and 13.8\u2009\u00b1\u20095.4\u00a0years at nusinersen initiation. One was lost to follow-up. At baseline, 6 patients used NIV and 8 used MIE. After 3\u00a0years, NIV use increased to 7\/27 and MIE to 14\/27. All 27 patients maintained oral intake and their BMI remained stable. ppFVC declined, most notably in year 3 (\u2212\u200913.5%,\n                    <jats:italic>p<\/jats:italic>\n                    \u2009&lt;\u20090.001). PCF, MVV, MIP, and MEP values remained stable or mildly improved, suggesting a slower decline or relative stabilization compared to natural progression.\n                  <\/jats:p>\n                  <jats:p>\n                    <jats:italic>Conclusion<\/jats:italic>\n                    : Nusinersen may help preserve respiratory muscle strength and delay the need for ventilatory support among pediatric patients with SMA types 2 and 3. Although ppFVC declined, stability in other parameters supports a potential respiratory benefit.\n                    <jats:table-wrap>\n                      <jats:table>\n                        <jats:tbody>\n                          <jats:tr>\n                            <jats:td colspan=\"2\">\n                              <jats:bold>What is Known:<\/jats:bold>\n                              \u2022 SMA causes progressive respiratory decline over time.\n                            <\/jats:td>\n                          <\/jats:tr>\n                          <jats:tr>\n                            <jats:td colspan=\"2\">\n                              <jats:bold>What is New:<\/jats:bold>\n                              \u2022 Nusinersen may preserve respiratory muscle strength in SMA.\n                            <\/jats:td>\n                          <\/jats:tr>\n                        <\/jats:tbody>\n                      <\/jats:table>\n                    <\/jats:table-wrap>\n                  <\/jats:p>","DOI":"10.1007\/s00431-025-06475-0","type":"journal-article","created":{"date-parts":[[2025,10,20]],"date-time":"2025-10-20T03:12:29Z","timestamp":1760929949000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Respiratory effects of nusinersen treatment in pediatric patients with spinal muscular atrophy types 2 and 3"],"prefix":"10.1007","volume":"184","author":[{"given":"Mika","family":"Rochman","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dar","family":"Shay Levanon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Israel","family":"Amirav","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moria","family":"Be\u2019er","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michal","family":"Cahal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omri","family":"Besor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aviva","family":"Fattal-Valevski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Moran","family":"Lavie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,10,20]]},"reference":[{"key":"6475_CR1","doi-asserted-by":"publisher","first-page":"88","DOI":"10.1186\/s13023-020-01367-y","volume":"15","author":"CA Wijngaarde","year":"2020","unstructured":"Wijngaarde CA, Veldhoen ES, Van Eijk RPA et al (2020) Natural history of lung function in spinal muscular atrophy. 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