Pre-existing anti-drug antibodies in Fabry disease show less affinity and inhibitory capacity for pegunigalsidase-alfa

Malte Lenders, Solvey Pollmann,Eva Brand

Molecular Genetics and Metabolism(2023)

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摘要
Pompe disease results from inherited deficiency of the enzyme acid alpha-glucosidase resulting in lysosomal accumulation of glycogen primarily in skeletal muscle. Reported is the first case in which a donor with late onset Pompe disease (LOPD) was successfully used for deceased donor liver and kidney transplantation. This case demonstrates co-operative transplant surgery and genetic medicine evaluation and risk estimation for donors with inherited metabolic disorders some of which may be suitable for donation of selected organs for transplantation.
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关键词
fabry disease,antibodies,pre-existing,anti-drug,pegunigalsidase-alfa
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