Article
Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.
Genetics in medicine : official journal of the American College of Medical Genetics - 1 Jan 2017
de Vries Juna M, Kuperus Esther, Hoogeveen-Westerveld Marianne, Kroos Marian A, Wens Stephan C A, Stok Merel, van der Beek Nadine A M E, Kruijshaar Michelle E, Rizopoulos Dimitris, van Doorn Pieter A, van der Ploeg Ans T, Pijnappel W W M Pim
Abstract excerpt
PURPOSE: To determine the effect of antibodies against recombinant human acid α-glucosidase (rhGAA) on treatment efficacy and safety, and to test whether the GAA genotype is involved in antibody formation. METHODS: We used enzyme-linked immunosorbent assay (ELISA) to determine anti-rhGAA antibody titers at baseline and at 6, 12, and 36 months of rhGAA treatment. We measured the capacity of antibodies to...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.