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The impact of interrupting enzyme replacement therapy in late-onset Pompe disease

BACKGROUND: Late-onset Pompe disease (LOPD) is a rare autosomal recessive disorder caused by mutations in the GAA gene, leading to progressive weakness of locomotor and respiratory muscles. Enzyme replacement therapy (ERT), administered every second week, has been proven to slow down disease progres...

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Detalhes bibliográficos
Publicado no:J Neurol
Main Authors: Wenninger, Stephan, Gutschmidt, Kristina, Wirner, Corinna, Einvag, Krisztina, Montagnese, Federica, Schoser, Benedikt
Formato: Artigo
Idioma:Inglês
Publicado em: Springer Berlin Heidelberg 2021
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7903209/
https://ncbi.nlm.nih.gov/pubmed/33625582
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00415-021-10475-z
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