{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T14:06:49Z","timestamp":1792159609253,"version":"4.4.0"},"reference-count":25,"publisher":"Massachusetts Medical Society","issue":"10","license":[{"start":{"date-parts":[[2021,3,11]],"date-time":"2021-03-11T00:00:00Z","timestamp":1615420800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"DOI":"10.13039\/100007013","name":"F. Hoffmann\u2013La Roche","doi-asserted-by":"publisher","award":["n\/a"],"award-info":[{"award-number":["n\/a"]}],"id":[{"id":"10.13039\/100007013","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2021,3,11]]},"DOI":"10.1056\/nejmoa2009965","type":"journal-article","created":{"date-parts":[[2021,2,24]],"date-time":"2021-02-24T17:01:16Z","timestamp":1614186076000},"page":"915-923","source":"Crossref","is-referenced-by-count":424,"title":["Risdiplam in Type 1 Spinal Muscular Atrophy"],"prefix":"10.1056","volume":"384","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4871-6692","authenticated-orcid":false,"given":"Giovanni","family":"Baranello","sequence":"first","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Basil T.","family":"Darras","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John W.","family":"Day","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas","family":"Deconinck","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrea","family":"Klein","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Riccardo","family":"Masson","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugenio","family":"Mercuri","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristy","family":"Rose","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muna","family":"El-Khairi","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marianne","family":"Gerber","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ksenija","family":"Gorni","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Omar","family":"Khwaja","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Heidemarie","family":"Kletzl","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renata S.","family":"Scalco","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy","family":"Seabrook","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paulo","family":"Fontoura","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laurent","family":"Servais","sequence":"additional","affiliation":[{"name":"From the Dubowitz Neuromuscular Centre, National Institute for Health Research Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health University College London, and Great Ormond Street Hospital Trust, London (G.B.), Roche Products, Welwyn Garden City (M.E.-K.), and the Muscular Dystrophy UK Oxford Neuromuscular Centre, Department of Paediatrics, University of Oxford, Oxford (L.S.) \u2014 all in the United Kingdom; the Developmental Neurology Unit, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan (G.B., R.M.), and the Pediatric Neurology Institution, Catholic University and Nemo Pediatrico, Fondazione Policlinico Gemelli IRCCS, Rome (E.M.); the Department of Neurology, Boston Children\u2019s Hospital, Harvard Medical School, Boston (B.T.D.); the Department of Neurology, Stanford University, Palo Alto, CA (J.W.D.); Centre de R\u00e9f\u00e9rence des Maladies Neuromusculaires, Queen Fabiola Children\u2019s University Hospital, Universit\u00e9 Libre de Bruxelles, Brussels (N.D.), the N..."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"150","reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(12)70061-3"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(95)90460-3"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbagrm.2016.12.008"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.11.6307"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2020.01.003"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000000741"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25101"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2008-2952K"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1186\/s13023-020-01367-y"},{"key":"e_1_3_5_12_2","unstructured":"Food and Drug Administration. Spinraza (nusinersen) prescribing information. 2016 (https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/209531lbl.pdf)."},{"key":"e_1_3_5_13_2","unstructured":"Food and Drug Administration. Zolgensma (onasemnogene abeparvovec-xioi) prescribing information. 2019 (https:\/\/www.fda.gov\/media\/126109\/download)."},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002445"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.8b00741"},{"key":"e_1_3_5_19_2","unstructured":"Evrysdi (risdiplam) prescribing information. Gene.com. 2020 (https:\/\/www.gene.com\/download\/pdf\/evrysdi_prescribing.pdf)."},{"key":"e_1_3_5_20_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41467-017-01559-4"},{"key":"e_1_3_5_21_2","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.447"},{"key":"e_1_3_5_22_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0139950"},{"key":"e_1_3_5_23_2","doi-asserted-by":"publisher","DOI":"10.1111\/bcp.13786"},{"key":"e_1_3_5_24_2","doi-asserted-by":"crossref","unstructured":"Bayley N. Bayley scales of infant and toddler development. 3rd edition. London: Psychological Corporation 2006.","DOI":"10.1037\/t14978-000"},{"key":"e_1_3_5_25_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_3_5_26_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(99)70016-8"}],"container-title":["New England Journal of Medicine"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.nejm.org\/doi\/pdf\/10.1056\/NEJMoa2009965","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,6,11]],"date-time":"2025-06-11T18:30:06Z","timestamp":1749666606000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.nejm.org\/doi\/10.1056\/NEJMoa2009965"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,3,11]]},"references-count":25,"journal-issue":{"issue":"10","published-print":{"date-parts":[[2021,3,11]]}},"alternative-id":["10.1056\/NEJMoa2009965"],"URL":"https:\/\/doi.org\/10.1056\/nejmoa2009965","relation":{},"ISSN":["0028-4793","1533-4406"],"issn-type":[{"value":"0028-4793","type":"print"},{"value":"1533-4406","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,3,11]]}}}