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The neuromuscular impact of symptomatic SMN restoration in a mouse model of spinal muscular atrophy

BACKGROUND: Significant advances in the development of SMN-restoring therapeutics have occurred since 2010 when very effective biological treatments were reported in mouse models of spinal muscular atrophy. As these treatments are applied in human clinical trials, there is pressing need to define qu...

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Bibliographic Details
Published in:Neurobiol Dis
Main Authors: Arnold, W. David, McGovern, Vicki L., Sanchez, Benjamin, Li, Jia, Corlett, Kaitlyn M., Kolb, Stephen J., Rutkove, Seward B., Burghes, Arthur H.
Format: Artigo
Language:Inglês
Published: 2015
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4724465/
https://ncbi.nlm.nih.gov/pubmed/26733414
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.nbd.2015.12.014
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