Zobrazeno 1 - 1
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pro vyhledávání: '"Sabina Kineen"'
Autor:
Daniel G. Bichet, Robert J. Hopkin, Patrício Aguiar, Sridhar R. Allam, Yin-Hsiu Chien, Roberto Giugliani, Staci Kallish, Sabina Kineen, Olivier Lidove, Dau-Ming Niu, Iacopo Olivotto, Juan Politei, Paul Rakoski, Roser Torra, Camilla Tøndel, Derralynn A. Hughes
Publikováno v:
Frontiers in Medicine, Vol 10 (2023)
ObjectiveFabry disease is a progressive disorder caused by deficiency of the α-galactosidase A enzyme (α-Gal A), leading to multisystemic organ damage with heterogenous clinical presentation. The addition of the oral chaperone therapy migalastat to
Externí odkaz:
https://doaj.org/article/95cf0962320c4c5da4b8e36cf0ea24fc
