First results of a head-to-head trial of pegunigalsidase alfa vs. agalsidase beta in Fabry disease: 2 year results of the phase 3 randomized, double-blind, BALANCE study

Eric Wallace, Wilcox Goker-Alpan, Bernat Holida,N. Longo,D. Hughes, P. Giraldo,M.J. Molnar, Hopkin Ortiz, C. Tøndel, A. Linhart, Jovanovic Deegan, M. Muriello, Kimonis Barshop,B. Vujkovac, A. Nowak,T. Geberhiwot, A. Pisani,D.P. Germain, I. Kantola, J. Knoll

Molecular Genetics and Metabolism(2023)

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摘要
The specific features of the microstructure of multicomponent solid solutions, based on relaxor ferroelectrics both unmodified and modified by barium, were observed. A reduction of tetragonal distortion in the unit cell in ceramics containing Ba, as well as an increase in the size of its crystallites (which is a consequence of this reduction), facilitates domain reorientation owing to an increase in their mobility. This is the main reason for the sharp increase in the relative permittivity εT33/ε0 of the poled samples of modified ceramics, and the variations of some parameters that characterize the piezoelectric response.
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关键词
fabry disease,agalsidase beta,pegunigalsidase alfa,head-to-head,double-blind
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