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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Pubblicato in:Neurobiol Dis
Autori principali: Arnold, W. David, McGovern, Vicki L., Sanchez, Benjamin, Li, Jia, Corlett, Kaitlyn M., Kolb, Stephen J., Rutkove, Seward B., Burghes, Arthur H.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2015
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Article
Accesso online: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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