Antibodies against recombinant human alpha-glucosidase do not seem to affect clinical outcome in childhood onset Pompe disease

BACKGROUND: Enzyme replacement therapy (ERT) with recombinant human alpha-glucosidase (rhGAA, alglucosidase alfa) has improved survival, motor outcomes, daily life activity and quality of life in Pompe patients. However, ERT in Pompe disease often induces formation of antibodies, which may reduce th...

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Detalles Bibliográficos
Autores principales: van Kooten, Harmke A., Ditters, Imke A. M., Hoogeveen-Westerveld, Marianne, Jacobs, Edwin H., van den Hout, Johanna M. P., van Doorn, Pieter A., Pijnappel, W. W. M. Pim, van der Ploeg, Ans T., van der Beek, Nadine A. M. E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8812154/
https://www.ncbi.nlm.nih.gov/pubmed/35109913
http://dx.doi.org/10.1186/s13023-022-02175-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8812154/
https://www.ncbi.nlm.nih.gov/pubmed/35109913
http://dx.doi.org/10.1186/s13023-022-02175-2

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