To assess phosphorylated neurofilament heavy chain (pNF-H) levels in individuals from nusinersen clinical trials with presymptomatic (most likely to develop Type I/II), infantile-onset (has or most likely to develop Type I/II) or later-onset SMA (has or most likely to develop Type II/III) and volunteers without SMA and investigate the association with motor function (MF) achievement in nusinersen-treated individuals with SMA.
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Sumner et al. (2019) studied this question.