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...
Main Authors: | W. Arnold, Vicki L. McGovern, Benjamin Sanchez, Jia Li, Kaitlyn M. Corlett, Stephen J. Kolb, Seward B. Rutkove, Arthur H. Burghes |
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Format: | Article |
Language: | English |
Published: |
Elsevier
2016-03-01
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Series: | Neurobiology of Disease |
Subjects: | |
Online Access: | http://www.sciencedirect.com/science/article/pii/S0969996115301170 |
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