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Primary outcomes were: safety and adverse events; secondary outcomes were survival, time to death\/&gt;16 hours\/day of ventilator support; motor outcomes; and maximum ulnar compound motor action potential amplitude. <jats:italic>Results:<\/jats:italic> A total of 245 AEs were observed in 35 of the 37 treated subjects (95%). Respiratory events accounted for 49% of all adverse events, resulting in 14 deaths. Survival was not significantly different between treated and untreated cohorts. <jats:italic>Discussion:<\/jats:italic> This trial provides evidence that, in infants with SMA type I, <jats:sc>L<\/jats:sc>\u2010carnitine\/VPA is ineffective at altering survival. The substantial proportion of infants reaching end\u2010points within 6 months of enrollment underscores the urgent need for pre\u2010symptomatic treatment in SMA type I. <jats:italic>Muscle Nerve<\/jats:italic> <jats:bold>57<\/jats:bold>: 193\u2013199, 2018<\/jats:p>","DOI":"10.1002\/mus.25776","type":"journal-article","created":{"date-parts":[[2017,8,18]],"date-time":"2017-08-18T17:20:44Z","timestamp":1503076844000},"page":"193-199","source":"Crossref","is-referenced-by-count":24,"title":["Clinical trial of L\u2010Carnitine and valproic acid in spinal muscular atrophy type I"],"prefix":"10.1002","volume":"57","author":[{"given":"Kristin J.","family":"Krosschell","sequence":"first","affiliation":[{"name":"Department of Physical Therapy and Human Movement Sciences, Feinberg School of Medicine Northwestern University Chicago Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John T.","family":"Kissel","sequence":"additional","affiliation":[{"name":"Department 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