Development of high sustained anti-drug antibody titers and corresponding clinical decline in a late-onset Pompe disease patient after 11+ years on enzyme replacement therapy

Development of high sustained anti-drug antibody titers and corresponding clinical decline in a late-onset Pompe disease patient after 11+ years on enzyme replacement therapy

A late-onset Pompe disease patient developed high sustained antibody titers (HSAT) of ≥51,200 after 11+ years on alglucosidase alfa and previous tolerance. There was a corresponding worsening of motor function and rise in urinary glucose tetrasaccharide (Glc4). Following immunomodulation therapy, HS...

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Bibliographic Details
Main Authors: Katherine H. Kim, Ankit K. Desai, Erika R. Vucko, Tracy Boggs, Priya S. Kishnani, Barbara K. Burton
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Genetics and Metabolism Reports
Subjects:
Pompe disease
Anti-drug antibody titers
Enzyme replacement therapy
Immune modulation therapy
Urine glucose tetrasaccharide (Glc4)
Online Access:http://www.sciencedirect.com/science/article/pii/S2214426923000277

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http://www.sciencedirect.com/science/article/pii/S2214426923000277

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