Effects of agalsidase-β shortage in Fabry disease

2015 ◽  
Vol 11 (10) ◽  
pp. 566-566
Keyword(s):  
Fabry Disease ◽  
Agalsidase Β

scholarly journals Effects of agalsidase-β administration on vascular function and blood pressure in familial Anderson–Fabry disease

2020 ◽  
Author(s):  
Cosimo Andrea Stamerra ◽  
Martina De Feo ◽  
Vanessa Castelli ◽  
Michele d’Angelo ◽  
Annamaria Cimini ◽  
...  
Keyword(s):  
Blood Pressure ◽  
Fabry Disease ◽  
Vascular Function ◽  
Agalsidase Β

scholarly journals Generation and Characterization of a Polyclonal Human Reference Antibody to Measure Anti-Drug Antibody Titers in Patients with Fabry Disease

2021 ◽  
Vol 22 (5) ◽  
pp. 2680
Author(s):  
Malte Lenders ◽  
David Scharnetzki ◽  
Ali Heidari ◽  
Daniele Di Iorio ◽  
Seraphine Valeska Wegner ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Serum Samples ◽  
Enzyme Replacement ◽  
Patients At Risk ◽  
Antibody Titers ◽  
Male Patients ◽  
The Individual ◽  
Agalsidase Α ◽  
Agalsidase Β

Male patients with Fabry disease (FD) are at high risk for the formation of antibodies to recombinant α-galactosidase A (AGAL), used for enzyme replacement therapy. Due to the rapid disease progression, the identification of patients at risk is highly warranted. However, currently suitable references and standardized protocols for anti-drug antibodies (ADA) determination do not exist. Here we generate a comprehensive patient-derived antibody mixture as a reference, allowing ELISA-based quantification of antibody titers from individual blood samples. Serum samples of 22 male patients with FD and ADAs against AGAL were pooled and purified by immune adsorption. ADA-affinities against agalsidase-α, agalsidase-β and Moss-AGAL were measured by quartz crystal microbalance with dissipation monitoring (QCM-D). AGAL-specific immune adsorption generated a polyclonal ADA mixture showing a concentration-dependent binding and inhibition of AGAL. Titers in raw sera and from purified total IgGs (r2 = 0.9063 and r2 = 0.8952, both p < 0.0001) correlated with the individual inhibitory capacities of ADAs. QCM-D measurements demonstrated comparable affinities of the reference antibody for agalsidase-α, agalsidase-β and Moss-AGAL (KD: 1.94 ± 0.11 µM, 2.46 ± 0.21 µM, and 1.33 ± 0.09 µM, respectively). The reference antibody allows the ELISA-based ADA titer determination and quantification of absolute concentrations. Furthermore, ADAs from patients with FD have comparable affinities to agalsidase-α, agalsidase-β and Moss-AGAL.

scholarly journals Sustained, Long-Term Renal Stabilization After 54 Months of Agalsidase β Therapy in Patients with Fabry Disease

2007 ◽  
Vol 18 (5) ◽  
pp. 1547-1557 ◽  
Author(s):  
Dominique P. Germain ◽  
Stephen Waldek ◽  
Maryam Banikazemi ◽  
David A. Bushinsky ◽  
Joel Charrow ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Agalsidase Β

scholarly journals Time to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry

2016 ◽  
Vol 53 (7) ◽  
pp. 495-502 ◽  
Author(s):  
Alberto Ortiz ◽  
Ademola Abiose ◽  
Daniel G Bichet ◽  
Gustavo Cabrera ◽  
Joel Charrow ◽  
...  
Keyword(s):  
Fabry Disease ◽  
Adult Patients ◽  
Treatment Benefit ◽  
Time To Treatment ◽  
Agalsidase Β

Differences between agalsidase α and agalsidase β in the treatment of Fabry disease

2017 ◽  
Vol 149 (6) ◽  
pp. 270
Author(s):  
Jordi Pérez-López ◽  
Alfonso Domínguez Gil-Hurlé ◽  
Mónica López Rodríguez
Keyword(s):  
Fabry Disease ◽  
Agalsidase Α ◽  
Agalsidase Β
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