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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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| Publicado no: | J Neurol |
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| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Springer Berlin Heidelberg
2021
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| Assuntos: | |
| 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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