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Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease

OBJECTIVE: We have recently reported on the pathology of the neuromuscular junction (NMJ) in Pompe disease, reflecting disruption of neuronal and muscle homeostasis as a result of glycogen accumulation. The aim of this study was to examine how the alteration of NMJ physiology contributes to Pompe di...

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Detalhes bibliográficos
Publicado no:Ann Neurol
Main Authors: Todd, Adrian G., McElroy, Jessica A., Grange, Robert W., Fuller, David D., Walter, Glenn A., Byrne, Barry J., Falk, Darin J.
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4520217/
https://ncbi.nlm.nih.gov/pubmed/25925726
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24433
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