B-cell Depletion and Immunomodulation Prior to Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha Glucosidase in Infantile Onset Pompe Disease

OBJECTIVES: Pompe disease is a progressive neuromuscular disorder due to acid alpha glucosidase (GAA) deficiency. Cross-reacting immunologic material (CRIM)-negative infants with null GAA mutations have the most severe phenotype and develop anti-GAA antibodies following exposure to enzyme replacemen...

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Hlavní autoři: Elder, Melissa E., Nayak, Sushrusha, Collins, Shelley W., Lawson, Lee Ann, Kelley, Jeffry S., Herzog, Roland W., Modica, Renee F., Lew, Judy, Lawrence, Robert M., Byrne, Barry J.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2013
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3981605/
https://ncbi.nlm.nih.gov/pubmed/23601496
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.jpeds.2013.03.002
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