Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions

BACKGROUND: Enzyme replacement therapy (ERT) with alglucosidase alfa is the treatment for patients with Pompe disease, a hereditary metabolic myopathy. Home-based ERT is unavailable in many countries because of the boxed warning alglucosidase alfa received due to the risk of infusion-associated reac...

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Detalles Bibliográficos
Autores principales: Ditters, Imke A. M., van Kooten, Harmke A., van der Beek, Nadine A. M. E., Hardon, Jacqueline F., Ismailova, Gamida, Brusse, Esther, Kruijshaar, Michelle E., van der Ploeg, Ans T., van den Hout, Johanna M. P., Huidekoper, Hidde H.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2023
Materias:
Original Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432339/
https://www.ncbi.nlm.nih.gov/pubmed/37326923
http://dx.doi.org/10.1007/s40259-023-00609-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432339/
https://www.ncbi.nlm.nih.gov/pubmed/37326923
http://dx.doi.org/10.1007/s40259-023-00609-2

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