Clinical trial of L-Carnitine and valproic acid in spinal muscular atrophy type I.
Kristin J KrosschellJohn T KisselElise L TownsendSarah D SimeoneRen Zhe ZhangSandra P ReynaThomas O CrawfordMary K SchrothGyula AcsadiPriya S KishnaniJürgen-Christoph Von Kleist-RetzowBarbara HeroGuy D'AnjouEdward C SmithBakri ElsheikhLouise R SimardThomas W PriorCharles B ScottBernard LasalleAi SakonjuBrunhilde WirthKathryn J Swobodanull nullPublished in: Muscle & nerve (2017)
This trial provides evidence that, in infants with SMA type I, L-carnitine/VPA is ineffective at altering survival. The substantial proportion of infants reaching end-points within 6 months of enrollment underscores the urgent need for pre-symptomatic treatment in SMA type I. Muscle Nerve 57: 193-199, 2018.