{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,23]],"date-time":"2026-10-23T23:29:13Z","timestamp":1792798153964,"version":"4.5.0"},"reference-count":20,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2019,12,4]],"date-time":"2019-12-04T00:00:00Z","timestamp":1575417600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Institut de Myologie","id":[{"id":"https:\/\/ror.org\/0270xt841","id-type":"ROR","asserted-by":"crossref"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Develop Med Child Neuro"],"published-print":{"date-parts":[[2020,3]]},"abstract":"<jats:sec>\n                    <jats:title>Aim<\/jats:title>\n                    <jats:p>To determine factors associated with acquisition of a sitting position in patients with spinal muscular atrophy type 1 (SMA1) treated with nusinersen.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Method<\/jats:title>\n                    <jats:p>\n                      Using data from the registry of patients with SMA1 treated with nusinersen, we compared the subgroups of sitters and non\u2010sitters after 14\u00a0months of therapy as a function of baseline level,\n                      <jats:italic>SMN2<\/jats:italic>\n                      copy number, age at treatment initiation, and improvement at 2 and 6\u00a0months post\u2010treatment initiation. We used Hammersmith Infant Neurological Examination, Section 2 (HINE\u20102) and Children\u2019s Hospital of Philadelphia Infant Test of Neuromuscular Disorders for motor evaluation.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Fifty children (22 females, 28 males), mean age 22\u00a0months (SD 20.7; range 2.5\u2013102.8mo) were treated. Data on sitting position acquisition were collected for 47 patients at month 14. Fifteen patients were able to sit unassisted; 11 of 15 had a baseline HINE\u20102 score of at least 2 points and 11 of 14 had an improvement over baseline of at least 2 points at month 6. Patients who improved by 2 or more points at month 6 were three times more likely to be sitters at month 14 than those who did not.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Interpretation<\/jats:title>\n                    <jats:p>High baseline motor function and improvement in HINE\u20102 score after 6\u00a0months of treatment are associated with the probability of acquiring a sitting position in patients with SMA1 treated with nusinersen.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>What this paper adds<\/jats:title>\n                    <jats:p>\n                      <jats:list list-type=\"bullet\">\n                        <jats:list-item>\n                          <jats:p>Fifteen of 47 patients with spinal muscular atrophy could sit unaided 14\u00a0months after treatment with nusinersen.<\/jats:p>\n                        <\/jats:list-item>\n                        <jats:list-item>\n                          <jats:p>\n                            The number of\n                            <jats:italic>SMN2<\/jats:italic>\n                            copies were not predictive of acquisition of a sitting position.\n                          <\/jats:p>\n                        <\/jats:list-item>\n                        <jats:list-item>\n                          <jats:p>Baseline condition and clinical response after 6\u00a0months of treatment were most predictive of sitting position acquisition.<\/jats:p>\n                        <\/jats:list-item>\n                      <\/jats:list>\n                    <\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/dmcn.14412","type":"journal-article","created":{"date-parts":[[2019,12,4]],"date-time":"2019-12-04T07:24:40Z","timestamp":1575444280000},"page":"310-314","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":42,"title":["Sitting in patients with spinal muscular atrophy type 1 treated with nusinersen"],"prefix":"10.1111","volume":"62","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-6447-6388","authenticated-orcid":false,"given":"Karolina","family":"Aragon\u2010Gawinska","sequence":"first","affiliation":[{"name":"Institute I\u2010motion Armand Trousseau Hospital Paris France"},{"name":"Department of Neurology Warsaw Medical University Warsaw Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aurore","family":"Daron","sequence":"additional","affiliation":[{"name":"Neuromuscular Reference Centre Citadelle Hospital Liege Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Ulinici","sequence":"additional","affiliation":[{"name":"Neuromuscular Reference Centre Citadelle Hospital Liege Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Vanden Brande","sequence":"additional","affiliation":[{"name":"Institute I\u2010motion Armand Trousseau Hospital Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andreea","family":"Seferian","sequence":"additional","affiliation":[{"name":"Institute I\u2010motion Armand Trousseau Hospital Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teresa","family":"Gidaro","sequence":"additional","affiliation":[{"name":"Institute I\u2010motion Armand Trousseau Hospital Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mariacristina","family":"Scoto","sequence":"additional","affiliation":[{"name":"Neuromuscular Centre UCL Great Ormond Street Institute of Child Health London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Deconinck","sequence":"additional","affiliation":[{"name":"Department of Paediatric Neurology and Neuromuscular Centre H\u00f4pital Universitaire des Enfants Reine Fabiola (HUDERF) ULB Brussels Belgium"},{"name":"Department of Paediatric Neurology Neuromuscular Reference Centre UZ Gent Ghent Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9270-4061","authenticated-orcid":false,"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"Institute I\u2010motion Armand Trousseau Hospital Paris France"},{"name":"Neuromuscular Reference Centre Citadelle Hospital Liege Belgium"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"SMA\u2010Registry Study 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report on the safety and efficacy of longer\u2010term treatment with nusinersen in infantile\u2010onset spinal muscular atrophy (SMA): updated results from the SHINE Study","volume":"92","author":"Finkel R","year":"2019","journal-title":"Neurology"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.2147\/TCRM.S172291"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.05.005"},{"key":"e_1_2_7_17_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-199001"},{"key":"e_1_2_7_18_1","doi-asserted-by":"publisher","DOI":"10.3233\/JND-190403"},{"issue":"15","key":"e_1_2_7_19_1","doi-asserted-by":"crossref","first-page":"S25.001","DOI":"10.1212\/WNL.92.15_supplement.S25.001","article-title":"Nusinersen in infants who initiate treatment in a presymptomatic stage of spinal muscular atrophy (SMA): interim efficacy and safety results from the Phase 2 NURTURE Study (S25.001)","volume":"92","author":"De Vivo 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