Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy
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DOI: 10.1016/j.neuron.2018.02.009
Abstract: Spinal muscular atrophy (SMA) is a common and oft-fatal pediatric neuromuscular disorder caused by insufficient SMN protein. Now, two clinical trials (Mendell et al., 2017; Finkel et al., 2017) demonstrate that restoring the protein is therapeutic, offering… read more here.
Keywords: smn protein; spinal muscular; muscular atrophy; augmenting smn ... See more keywords