Pompe disease in adulthood: effects of antibody formation on enzyme replacement therapy.

Juna M de VriesEsther KuperusMarianne Hoogeveen-WesterveldMarian A KroosStephan C A WensMerel StokNadine A M E van der BeekMichelle E KruijshaarDimitris RizopoulosPieter A van DoornAns T van der PloegW W M Pim Pijnappel

Published in: Genetics in medicine : official journal of the American College of Medical Genetics (2016)

In adults with Pompe disease, antibody formation does not interfere with rhGAA efficacy in the majority of patients, is associated with IARs, and may be attenuated by the IVS1/delex18 GAA genotype.Genet Med 19 1, 90-97.

Keyphrases

replacement therapy
smoking cessation
end stage renal disease
late onset
ejection fraction
newly diagnosed
chronic kidney disease
peritoneal dialysis
depressive symptoms
patient reported outcomes
early life