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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… Click to show full 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 new treatment options and renewed hope to SMA patients.
Keywords:
smn protein;
spinal muscular;
muscular atrophy;
augmenting smn
Journal Title: Neuron
Year Published: 2018
Link to full text (if available)
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Journal Title: Neuron
Year Published: 2018
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!