Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

Deficiency of acid alpha glucosidase (GAA) causes Pompe disease, which is usually fatal if onset occurs in infancy. Patients synthesize a non-functional form of GAA or are unable to form native enzyme. Enzyme replacement therapy with recombinant human GAA (rhGAA) prolongs survival in infantile Pompe...

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Detalhes bibliográficos
Main Authors: Kishnani, Priya S., Goldenberg, Paula C., DeArmey, Stephanie L., Heller, James, Benjamin, Danny, Young, Sarah, Bali, Deeksha, Smith, Sue Ann, Li, Jennifer S., Mandel, Hanna, Koeberl, Dwight, Rosenberg, Amy, Chen, Y-T
Formato: Artigo
Idioma:Inglês
Publicado em: 2010
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3721340/
https://ncbi.nlm.nih.gov/pubmed/19775921
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2009.08.003
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