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

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
Main Authors: W. Arnold, Vicki L. McGovern, Benjamin Sanchez, Jia Li, Kaitlyn M. Corlett, Stephen J. Kolb, Seward B. Rutkove, Arthur H. Burghes
Format: Article
Language:English
Published: Elsevier 2016-03-01
Series:Neurobiology of Disease
Subjects:
Spinal muscular atrophy
Biomarker
Survival motor neuron
Antisense oligonucleotide
Electrical impedance myography
Motor unit number estimation
Online Access:http://www.sciencedirect.com/science/article/pii/S0969996115301170

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http://www.sciencedirect.com/science/article/pii/S0969996115301170

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