{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T02:19:12Z","timestamp":1790475552096,"version":"4.1.0"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2020,8,10]],"date-time":"2020-08-10T00:00:00Z","timestamp":1597017600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Muscle and Nerve"],"published-print":{"date-parts":[[2020,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a retrospective study describing our centers' experiences in treating SMA1 patients with combination therapy.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Five children received nusinersen and onasemnogene abeparvovec\u2010xioi (onasemnogene). Four were receiving nusinersen prior to onasemnogene. Nusinersen was continued in three. Marked liver enzyme elevations resulted in prolonged corticosteroid treatment in two patients with hospitalization and liver biopsy in one; milder liver enzyme elevations were noted in the other two. One patient received onasemnogene first, and then nusinersen. No adverse effects were noted. All patients improved.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Combination molecular therapy is tolerated in SMA1 patients. Further studies are needed to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy. Prolonged corticosteroid use and liver toxicity monitoring may be necessary with onasemnogene therapy.<\/jats:p><\/jats:sec>","DOI":"10.1002\/mus.27034","type":"journal-article","created":{"date-parts":[[2020,7,25]],"date-time":"2020-07-25T12:58:51Z","timestamp":1595681931000},"page":"550-554","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":79,"title":["Combination molecular therapies for type 1 spinal muscular atrophy"],"prefix":"10.1002","volume":"62","author":[{"given":"Yohei","family":"Harada","sequence":"first","affiliation":[{"name":"Department of Neurology University of Arkansas for Medical Sciences  Little Rock Arkansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vamshi K.","family":"Rao","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Pediatrics Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kapil","family":"Arya","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Pediatrics Arkansas Children's Hospital, University of Arkansas for Medical Sciences  Little Rock Arkansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nancy L.","family":"Kuntz","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Pediatrics Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christine J.","family":"DiDonato","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Pediatrics Ann and Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine  Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Galia","family":"Napchan\u2010Pomerantz","sequence":"additional","affiliation":[{"name":"Department of Pediatric Pulmonology Joe DiMaggio Children's Hospital  Hollywood Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amit","family":"Agarwal","sequence":"additional","affiliation":[{"name":"Division of Pulmonology, Department of Pediatrics Arkansas Children's Hospital, University of Arkansas for Medical Sciences  Little Rock Arkansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vikki","family":"Stefans","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Physical Medicine and Rehabilitation Arkansas Children's Hospital, University of Arkansas for Medical Sciences  Little Rock Arkansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9453-9311","authenticated-orcid":false,"given":"Masahisa","family":"Katsuno","sequence":"additional","affiliation":[{"name":"Department of Neurology Nagoya University Graduate School of Medicine  Nagoya Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3065-3956","authenticated-orcid":false,"given":"Aravindhan","family":"Veerapandiyan","sequence":"additional","affiliation":[{"name":"Division of Neurology, Department of Pediatrics Arkansas Children's Hospital, University of Arkansas for Medical Sciences  Little Rock Arkansas USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,8,10]]},"reference":[{"key":"e_1_2_9_2_1","unstructured":"U.S. Food and Drug Administration. AveXis Inc Zolgensma (onasemnogene abeparvovec\u2010xioi). [online].https:\/\/www.fda.gov\/vaccines-blood-biologics\/zolgensma. Accessed July 19 2020."},{"key":"e_1_2_9_3_1","unstructured":"U.S. Food and Drug Administration USFaD. SPINRAZA (nusinersen) injection for intrathecal use. [online].https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2016\/209531lbl.pdf. Accessed July 19 2020."},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1080\/14656566.2019.1704732"},{"key":"e_1_2_9_5_1","unstructured":"VeerapandiyanA.Spinal muscular atrophy. MedLink Neurology. July 5 2019. MedLink Corporation.2019."},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40263-019-00656-w"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)31408-8"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_2_9_9_1","first-page":"90","article-title":"AVXS\u2010101 phase 3 study in spinal muscular atrophy type 1","volume":"60","author":"Day JW","year":"2019","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2009.11.014"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2016.10.002"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2020.05.044"},{"key":"e_1_2_9_14_1","unstructured":"DayJW ChiribogaCA CrawfordTO DarrasBT FinkelR ConnollyAM. Onasemnogene Abeparvovec gene therapy for spinal muscular atrophy type 1: completed phase 3 US study (STR1VE) efficacy and safety. Muscular Dystrophy Association (MDA) Clinical and Scientific Conference2020."},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000008207"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2019-anzan.58"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pediatrneurol.2019.06.007"},{"key":"e_1_2_9_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.omtm.2017.11.007"},{"key":"e_1_2_9_19_1","unstructured":"ChandD FinkelRS MercuriE MassonR ParsonsJ KleynA et al. Intravenous Onasemnogene Abeparvovec for spinal muscular atrophy: integrated safety report. Muscular Dystrophy Association (MDA) Clinical and Scientific Conference2020."},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1710504"}],"container-title":["Muscle &amp; Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.wiley.com\/onlinelibrary\/tdm\/v1\/articles\/10.1002%2Fmus.27034","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27034","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27034","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27034","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,5]],"date-time":"2023-09-05T16:44:29Z","timestamp":1693932269000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27034"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,8,10]]},"references-count":19,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2020,10]]}},"alternative-id":["10.1002\/mus.27034"],"URL":"https:\/\/doi.org\/10.1002\/mus.27034","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2020,8,10]]},"assertion":[{"value":"2020-03-03","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-07-21","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2020-08-10","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}