Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent

Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA approved therapy for Pompe disease. Without ERT, severely affected individuals (early onset) succumb to the disease within 2 years of life. A spectrum of disease severity and progression exists dependi...

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Главные авторы: Nayak, Sushrusha, Doerfler, Phillip A., Porvasnik, Stacy L., Cloutier, Denise D., Khanna, Richie, Valenzano, Ken J., Herzog, Roland W., Byrne, Barry J.
Формат: Artigo
Язык:Inglês
Опубликовано: Public Library of Science 2014
Предметы:
Research Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC4045583/
https://ncbi.nlm.nih.gov/pubmed/24897114
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0098336
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