Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis
Sign Up to like & getrecommendations! Published in 2020 at "ESC Heart Failure"
DOI: 10.1002/ehf2.12647
Abstract: Long‐term treatment effect studies in large female Fabry patient groups are challenging to design because of phenotype heterogeneity and lack of appropriate comparison groups, and have not been reported. We compared long‐term cardiomyopathy and kidney… read more here.
Keywords: cardiomyopathy kidney; treatment; agalsidase beta; female fabry ... See more keywords
Fabry disease under enzyme replacement therapy—new insights in efficacy of different dosages
Sign Up to like & getrecommendations! Published in 2018 at "Nephrology Dialysis Transplantation"
DOI: 10.1093/ndt/gfx319
Abstract: Background Fabry patients on reduced dose of agalsidase-beta or after switch to agalsidase-alfa show a decline in estimated glomerular filtration rate (eGFR) and an increase of the Mainz Severity Score Index. Methods In this prospective… read more here.
Keywords: switch agalsidase; group; agalsidase; agalsidase beta ... See more keywords
A phase II, multicenter, open-label trial to evaluate the safety and efficacy of ISU303 (Agalsidase beta) in patients with Fabry disease
Sign Up to like & getrecommendations! Published in 2022 at "Medicine"
DOI: 10.1097/md.0000000000030345
Abstract: Background: Fabry disease (FD) is caused by a deficiency in the activity of the lysosomal enzyme, α-galactosidase A (α-Gal A), which leads to globotriaosylceramide (Gb3) deposition in multiple tissues. The current management of FD is… read more here.
Keywords: safety; gb3; plasma urine; efficacy ... See more keywords
Plain language summary of a study looking at heart muscle thickness and kidney function in women with Fabry disease who received agalsidase beta treatment.
Sign Up to like & getrecommendations! Published in 2022 at "Future cardiology"
DOI: 10.2217/fca-2022-0047
Abstract: WHAT IS FABRY DISEASE & WHAT IS THIS STUDY ABOUT? Fabry disease is a rare genetic condition that affects many different cells and organs in the body. People have Fabry disease when they inherit a… read more here.
Keywords: disease; heart; agalsidase beta; treatment ... See more keywords
Assessment and impact of dose escalation on anti-drug antibodies in Fabry disease
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DOI: 10.3389/fimmu.2022.1024963
Abstract: Enzyme replacement therapy (ERT) with recombinant α-galactosidase A (AGAL) can lead to the formation of neutralizing anti-drug antibodies (ADA), which significantly limit treatment efficacy in patients with Fabry disease (FD). The effects of dose escalation… read more here.
Keywords: dose escalation; drug antibodies; escalation; anti drug ... See more keywords