Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: clinical evidence of respiratory plasticity

Pompe disease is an inherited disorder due to a mutation in the gene that encodes acid α-glucosidase (GAA). Children with infantile-onset Pompe disease develop progressive hypotonic weakness and cardiopulmonary insufficiency that may eventually require mechanical ventilation (MV). Our team conducted...

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Detalhes bibliográficos
Publicado no:Exp Neurol
Main Authors: Smith, Barbara K., Martin, A. Daniel, Lawson, Lee Ann, Vernot, Valerie, Marcus, Jordan, Islam, Saleem, Shafi, Nadeem, Corti, Manuela, Collins, Shelley W., Byrne, Barry J.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5178134/
https://ncbi.nlm.nih.gov/pubmed/27453480
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.expneurol.2016.07.013
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