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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Bibliografische gegevens
Gepubliceerd in:J Neurol
Hoofdauteurs: Wenninger, Stephan, Gutschmidt, Kristina, Wirner, Corinna, Einvag, Krisztina, Montagnese, Federica, Schoser, Benedikt
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Springer Berlin Heidelberg 2021
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Original Communication
Online toegang: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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