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Introduction: The aim of this study was to determine the safety and therapeutic potential of L‐carnitine and valproic acid (VPA) in infants with spinal muscular atrophy (SMA). Methods: Our investigation was an open‐label phase 2 multicenter trial of L‐carnitine and VPA in infants with SMA type I with retrospective comparison to an untreated, matched cohort. Primary outcomes were: safety and adverse...
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