{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,8,22]],"date-time":"2025-08-22T04:58:01Z","timestamp":1755838681523,"version":"3.38.0"},"reference-count":0,"publisher":"University of Toronto Press Inc. (UTPress)","issue":"1","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Clinical and Investigative Medicine"],"published-print":{"date-parts":[[2018,3,27]]},"abstract":"<jats:p> In their landmark paper, Mendell et al. show that infants with spinal muscular atrophy (SMA) reached important motor milestones and survived longer when treated with AVXS-101 (AveXis), a viral vector containing DNA encoding the survival of motor neuron protein (SMN). Patients not only crawled, stood and walked independently, but learned to speak. These results are very encouraging for patients with SMA and offer hope for pediatric and adult patients with other types of motor neuron diseases. <\/jats:p>","DOI":"10.25011\/cim.v41i1.29461","type":"journal-article","created":{"date-parts":[[2018,3,30]],"date-time":"2018-03-30T09:40:32Z","timestamp":1522402832000},"page":"E31-E33","source":"Crossref","is-referenced-by-count":4,"title":["CIM Journal Club: Gene therapy for spinal muscular atrophy Comment on Mendell <i>et al.<\/i> N Engl J Med 2017;377:1713-22"],"prefix":"10.3138","volume":"41","author":[{"given":"Raphael","family":"Schneider","sequence":"first","affiliation":[{"name":"Clinical Investigator Program and Eliot Phillipson Clinician-Scientist Training Program, University of Toronto, Toronto, Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1696","container-title":["Clinical and Investigative Medicine"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/cimonline.ca\/index.php\/cim\/article\/download\/29461\/21949","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/utppublishing.com\/doi\/pdf\/10.25011\/cim.v41i1.29461","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,3,13]],"date-time":"2025-03-13T18:14:56Z","timestamp":1741889696000},"score":1,"resource":{"primary":{"URL":"https:\/\/utppublishing.com\/doi\/10.25011\/cim.v41i1.29461"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,3,27]]},"references-count":0,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2018,3,27]]}},"alternative-id":["10.25011\/cim.v41i1.29461"],"URL":"https:\/\/doi.org\/10.25011\/cim.v41i1.29461","relation":{},"ISSN":["1488-2353"],"issn-type":[{"type":"electronic","value":"1488-2353"}],"subject":[],"published":{"date-parts":[[2018,3,27]]}}}