{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T02:19:07Z","timestamp":1790475547990,"version":"4.1.0"},"reference-count":10,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,7,31]],"date-time":"2021-07-31T00:00:00Z","timestamp":1627689600000},"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":[[2021,10]]},"abstract":"Abstract<\/jats:title>\n \n Introduction\/Aims<\/jats:title>\n There are currently three medications approved for spinal muscular atrophy (SMA), but the use of these medications in combination has not been well described.<\/jats:p>\n <\/jats:sec>\n \n Methods<\/jats:title>\n This is a retrospective report of four cases of SMA treated with dual onasemnogene and risdiplam therapy at our institution.<\/jats:p>\n <\/jats:sec>\n \n Results<\/jats:title>\n Following onasemnogene therapy, all four patients experienced a perceived plateau of therapeutic benefit, at which time daily risdiplam was started. Transient fatigue and weakness was seen in two patients following risdiplam initiation, but this resolved within 1\u2009mo. One patient was hospitalized with severe acute respiratory syndrome coronavirus\u20102 (SARS\u2010CoV\u20102) and post\u2010viral pneumonia, weeks following risdiplam initiation. No other adverse effects related to onasemnogene and risdiplam combination therapy were identified and all patients experienced objective and subjective improvement.<\/jats:p>\n <\/jats:sec>\n \n Discussion<\/jats:title>\n Combination therapy with onasemnogene and risdiplam in patients with SMA appears to be well\u2010tolerated. Further large prospective trials are needed to determine whether dual therapy is more efficacious than monotherapy, and to identify rare adverse events that may occur with the use of combination therapy.<\/jats:p>\n <\/jats:sec>","DOI":"10.1002\/mus.27375","type":"journal-article","created":{"date-parts":[[2021,7,21]],"date-time":"2021-07-21T00:51:22Z","timestamp":1626828682000},"page":"487-490","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":49,"title":["Combination therapy with onasemnogene and risdiplam in spinal muscular atrophy type 1"],"prefix":"10.1002","volume":"64","author":[{"given":"Kelly F.","family":"Oechsel","sequence":"first","affiliation":[{"name":"Department of Neurology Wake Forest School of Medicine Winston\u2010Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael S.","family":"Cartwright","sequence":"additional","affiliation":[{"name":"Department of Neurology Wake Forest School of Medicine Winston\u2010Salem North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2021,7,31]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(16)31408-8"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_2_8_4_1","unstructured":"ZOLGENSMA | FDA. U.S. Food and Drug Administration. Updated March 16 2021.https:\/\/www.fda.gov\/vaccines-blood-biologics\/zolgensma. Accessed April 10 2021."},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1706198"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1021\/acs.jmedchem.8b00741"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/prp2.447"},{"key":"e_1_2_8_8_1","unstructured":"Risdiplam | FDA. U.S. Food and Drug Administration. Updated August 2020.https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2020\/213535s000lbl.pdf. Accessed April 10 2021."},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2012.05.067"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.nmd.2017.06.414"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2009965"}],"container-title":["Muscle & Nerve"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27375","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/mus.27375","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/mus.27375","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,8,25]],"date-time":"2023-08-25T10:43:21Z","timestamp":1692960201000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/mus.27375"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,7,31]]},"references-count":10,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2021,10]]}},"alternative-id":["10.1002\/mus.27375"],"URL":"https:\/\/doi.org\/10.1002\/mus.27375","archive":["Portico"],"relation":{},"ISSN":["0148-639X","1097-4598"],"issn-type":[{"value":"0148-639X","type":"print"},{"value":"1097-4598","type":"electronic"}],"subject":[],"published":{"date-parts":[[2021,7,31]]},"assertion":[{"value":"2021-05-27","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-07-18","order":1,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2021-07-31","order":2,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}