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An individually, modified approach to desensitize infants and young children with Pompe disease, and significant reactions to alglucosidase alfa infusions

PURPOSE: Pompe disease (PD) is a progressive metabolic myopathy for which the only available treatment is alglucosidase alfa (Myozyme®). Enzyme replacement therapy (ERT) has improved ventilator-free survival, and cardiac and motor functions in patients with infantile PD. However, for an adequate res...

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Detalhes bibliográficos
Main Authors: El-Gharbawy, Areeg H., Mackey, Joanne, DeArmey, Stephanie, Westby, Greg, Grinnell, Sherry G., Malovrh, Peggy, Conway, Robert, Kishnani, Priya S.
Formato: Artigo
Idioma:Inglês
Publicado em: 2011
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3711228/
https://ncbi.nlm.nih.gov/pubmed/21802969
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.07.004
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