Journal
NEURON
Volume 97, Issue 5, Pages 1001-1003Publisher
CELL PRESS
DOI: 10.1016/j.neuron.2018.02.009
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Funding
- NINDS NIH HHS [R56 NS104218, R21 NS099921] Funding Source: Medline
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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 the protein is therapeutic, offering new treatment options and renewed hope to SMA patients.
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