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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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| Опубликовано в: : | J Neurol |
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| Главные авторы: | , , , , , |
| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
Springer Berlin Heidelberg
2021
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| Предметы: | |
| Online-ссылка: | 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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