Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation

Patients with Pompe disease have realized significant medical benefits due to enzyme replacement therapy (ERT) infusions with alglucosidase alfa. However, regular infusions are time-consuming. Utilizing recommended infusion rates, infusion duration is 3 hours 45 minutes for a patient receiving the s...

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Veröffentlicht in:Mol Genet Metab
Hauptverfasser: Desai, Ankit K., Walters, Crista K., Cope, Heidi L., Kazi, Zoheb B., DeArmey, Stephanie M., Kishnani, Priya S.
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2017
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5808871/
https://ncbi.nlm.nih.gov/pubmed/29289479
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2017.12.435
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