Response to “Oral Chaperone Therapy Migalastat for the Treatment of Fabry Disease: Potentials and Pitfalls of Real‐World Data”

Volume: 106, Issue: 5, Pages: 927 - 928
Published: Jul 12, 2019
Abstract
We thank Dr Körver and colleagues for their interest in our work.1 We absolutely agree that validity of current amenability criteria arguably might still be subject to discussion. From the clinical viewpoint, the cutoff values of 1.2-fold relative and 3% absolute increase in alpha galactosidase activity chosen in the recent multicenter trials might be questionable particularly in patients with polymorphism-like genotypes, such as the benign...
Paper Details
Title
Response to “Oral Chaperone Therapy Migalastat for the Treatment of Fabry Disease: Potentials and Pitfalls of Real‐World Data”
Published Date
Jul 12, 2019
Volume
106
Issue
5
Pages
927 - 928
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