{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,10,18]],"date-time":"2025-10-18T10:45:06Z","timestamp":1760784306561,"version":"3.37.3"},"reference-count":23,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2017,7,28]],"date-time":"2017-07-28T00:00:00Z","timestamp":1501200000000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Clin Drug Investig"],"published-print":{"date-parts":[[2017,9]]},"DOI":"10.1007\/s40261-017-0557-5","type":"journal-article","created":{"date-parts":[[2017,7,28]],"date-time":"2017-07-28T12:04:18Z","timestamp":1501243458000},"page":"807-817","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":20,"title":["Nusinersen: The First Option Beyond Supportive Care for Spinal Muscular Atrophy"],"prefix":"10.1007","volume":"37","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0218-3325","authenticated-orcid":false,"given":"Vikas","family":"Maharshi","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shazia","family":"Hasan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2017,7,28]]},"reference":[{"key":"557_CR1","doi-asserted-by":"crossref","first-page":"15","DOI":"10.1111\/joa.12083","volume":"224","author":"M Shababi","year":"2014","unstructured":"Shababi M, Lorson CL, Rudnik-Schoneborn SS. Spinal muscular atrophy: a motor neuron disorder or a multi-organ disease? J. Anat. 2014;224:15\u201328.","journal-title":"J. Anat"},{"key":"557_CR2","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1007\/s13311-015-0337-y","volume":"12","author":"Constantin d\u2019Ydewalle","year":"2015","unstructured":"d\u2019Ydewalle Constantin, Sumner Charlotte J. Spinal muscular atrophy therapeutics: where do we stand? Neurotherapeutics. 2015;12:303\u201316.","journal-title":"Neurotherapeutics"},{"key":"557_CR3","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1002\/mus.24497","volume":"51","author":"WD Arnold","year":"2015","unstructured":"Arnold WD, Kassar D, Kissel JT. Spinal muscular atrophy: diagnosis and management in a new therapeutic era. Muscle Nerve. 2015;51:157\u201367.","journal-title":"Muscle Nerve"},{"key":"557_CR4","doi-asserted-by":"publisher","DOI":"10.1007\/s40265-017-0711-7","author":"SM Hoy","year":"2017","unstructured":"Hoy SM. Nusinersen: first global approval. Drugs. 2017. doi: 10.1007\/s40265-017-0711-7 .","journal-title":"Drugs"},{"key":"557_CR5","unstructured":"Sullivan DL. Spinraza spinal muscular atrophy. Washington state health care authority. 2017. https:\/\/www.hca.wa.gov\/assets\/program\/dur-spinraza-2017-2-22.pdf . Accessed 31 Mar 2017."},{"key":"557_CR6","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1515\/tnsci-2017-0001","volume":"8","author":"EW Ottesen","year":"2017","unstructured":"Ottesen EW. ISS-N1 makes the first FDA approved drug for spinal muscular atrophy. Transl Neurosci. 2017;8:1\u20136.","journal-title":"Transl Neurosci"},{"key":"557_CR7","doi-asserted-by":"publisher","DOI":"10.1038\/nn.4508","author":"DR Corey","year":"2017","unstructured":"Corey DR. Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy. Nat Neurosci. 2017. doi: 10.1038\/nn.4508 .","journal-title":"Nat Neurosci"},{"key":"557_CR8","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1089\/nat.2017.0665","volume":"27","author":"A-R Annemieke","year":"2017","unstructured":"Annemieke A-R. FDA approval of nusinersen for spinal muscular atrophy makes 2016 the year of splice modulating oligonucleotides. Nucleic Acid Therap. 2017;27:67\u20139.","journal-title":"Nucleic Acid Therap"},{"key":"557_CR9","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2017.52","author":"K Talbot","year":"2017","unstructured":"Talbot K, Tizzano EF. The clinical landscape for SMA in a new therapeutic era. Gene Therapy. 2017. doi: 10.1038\/gt.2017.52 .","journal-title":"Gene Therapy"},{"key":"557_CR10","unstructured":"FDA approves first drug for spinal muscular atrophy. FDA news release. 2016. https:\/\/www.fda.gov\/NewsEvents\/Newsroom\/PressAnnouncements\/ucm534611.htm . Accessed 3 Apr 2017."},{"key":"557_CR11","unstructured":"Approval package for: application number 209531Orig1s000. Center for drug evaluation and research. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000OtherR.pdf . Accessed 31 Mar 2017."},{"key":"557_CR12","unstructured":"Nusinersen prescribing information. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/209531lbl.pdf . Accessed 23 Mar 2017."},{"key":"557_CR13","unstructured":"Pharmacology review(s). http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000PharmR.pdf . Accessed 23 Mar 2017."},{"key":"557_CR14","unstructured":"Clinical pharmacology and biopharmaceutics review(s). Center for drug evaluation and research. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000ClinPharmR.pdf . Accessed 23 Mar 2017."},{"key":"557_CR15","unstructured":"IUPAC. Compendium of chemical terminology, 2nd ed. (the Gold Book). Compiled by A.D. McNaught and A. Wilkinson. Blackwell, Oxford (1997) (XML online corrected version: https:\/\/goldbook.iupac.org\/html\/N\/NT06911.html , last updated 2014-02-24)."},{"issue":"10063","key":"557_CR16","doi-asserted-by":"crossref","first-page":"3017","DOI":"10.1016\/S0140-6736(16)31408-8","volume":"388","author":"RS Finkel","year":"2016","unstructured":"Finkel RS, Chiriboga CA, Vajsar J, Day JW, Montes J, De Vivo DC, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2 open label, dose-escalation study. Lancet. 2016;388(10063):3017\u201326.","journal-title":"Lancet"},{"key":"557_CR17","doi-asserted-by":"crossref","unstructured":"Finkel RS. Primary efficacy and safety results from the phase 3 ENDEAR study of nusinersen in infants diagnosed with spinal muscular atrophy (SMA). 43rd annual congress of the British Paediatric Neurology Association. 2017. http:\/\/phx.corporate-ir.net\/External . Accessed 31 Mar 2017.","DOI":"10.1016\/j.ejpn.2017.04.1219"},{"key":"557_CR18","unstructured":"Other review(s). Center for drug evaluation and research. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000OtherR.pdf . Accessed 23 Mar 2017."},{"key":"557_CR19","doi-asserted-by":"crossref","first-page":"890","DOI":"10.1212\/WNL.0000000000002445","volume":"86","author":"CA Chiriboga","year":"2016","unstructured":"Chiriboga CA, Swoboda KJ, Darras BT, Iannaccone ST, Montes J, De Vivo DC, et al. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology. 2016;86:890\u20137.","journal-title":"Neurology"},{"key":"557_CR20","unstructured":"Summary review. Center for drug evaluation and research. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000SumR.pdf . Accessed 23 Mar 2017."},{"key":"557_CR21","unstructured":"Biogen. About Spinraza (homepage on internet). Cambridge: Biogen Inc.; 2017. https:\/\/www.spinraza.com\/en_us\/home\/about-spinraza.html . Accessed 10 July 2017."},{"key":"557_CR22","unstructured":"Medical review(s). Center for drug evaluation and research. http:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/nda\/2016\/209531Orig1s000MedR.pdf . Accessed 23 Mar 2017."},{"key":"557_CR23","unstructured":"Ozkaya O. CureSMA to discuss Spinraza use, high cost and insurance coverage in webinar set for Tuesday. SMA News Today. 13 Feb 2017. https:\/\/smanewstoday.com\/2017\/02\/13\/webinar-access-spinraza\/ Accessed 7 Apr 2017."}],"container-title":["Clinical Drug Investigation"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s40261-017-0557-5\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40261-017-0557-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s40261-017-0557-5.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,10,1]],"date-time":"2019-10-01T15:36:55Z","timestamp":1569944215000},"score":1,"resource":{"primary":{"URL":"http:\/\/link.springer.com\/10.1007\/s40261-017-0557-5"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,7,28]]},"references-count":23,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2017,9]]}},"alternative-id":["557"],"URL":"https:\/\/doi.org\/10.1007\/s40261-017-0557-5","relation":{},"ISSN":["1173-2563","1179-1918"],"issn-type":[{"type":"print","value":"1173-2563"},{"type":"electronic","value":"1179-1918"}],"subject":[],"published":{"date-parts":[[2017,7,28]]}}}