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Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study.

Stephen J KolbChristopher S CoffeyJon W YankeyKristin KrosschellWilliam David ArnoldSeward B RutkoveKathryn J SwobodaSandra P ReynaAi SakonjuBasil T DarrasRichard ShellNancy KuntzDiana CastroSusan T IannacconeJulie ParsonsAnne M ConnollyClaudia A ChiribogaCraig McDonaldW Bryan BurnetteKlaus WernerMathula ThangarajhPerry B ShiehErika FinangerMerit E CudkowiczMichelle M McGovernD Elizabeth McNeilRichard FinkelEdward KayeAllison KingsleySamantha R RenuschVicki L McGovernXueqian WangPhillip G ZaworskiThomas W PriorArthur H M BurghesAmy BartlettJohn T Kisselnull null
Published in: Annals of clinical and translational neurology (2016)
By the time infants were recruited and presented for the baseline visit, SMA infants had reduced motor function compared to controls. Ulnar CMAP, EIM, blood SMN mRNA levels, and serum protein analytes were able to distinguish between cohorts at the enrollment visit.
Keyphrases
  • small molecule