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Augmenting the SMN Protein to Treat Infantile Spinal Muscular Atrophy.

Jeong-Ki Kim, Umrao R Monani
Neuron 2018 March 8
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 new treatment options and renewed hope to SMA patients.

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