Are Anti-rhGAA Antibodies a Determinant of Treatment Outcome in Adults with Late-Onset Pompe Disease? A Systematic Review

Background: Pompe disease is a lysosomal storage disease characterised by skeletal and respiratory muscle weakness. Since 2006, enzyme replacement therapy (ERT) with alglucosidase alfa has been available. ERT significantly improves the prognosis of patients with Pompe disease. The effect of high ant...

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Detalles Bibliográficos
Autores principales: Ditters, Imke A. M., van Kooten, Harmke A., van der Beek, Nadine A. M. E., van der Ploeg, Ans T., Huidekoper, Hidde H., van den Hout, Johanna M. P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10526476/
https://www.ncbi.nlm.nih.gov/pubmed/37759814
http://dx.doi.org/10.3390/biom13091414

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10526476/
https://www.ncbi.nlm.nih.gov/pubmed/37759814
http://dx.doi.org/10.3390/biom13091414

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