Listar por autor "Rakoski, Paul"

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    • Consensus recommendations for the treatment and management of patients with Fabry disease on migalastat : a modified Delphi study 

      Bichet, Daniel G.; Hopkin, Robert J.; Aguiar, Patrício; Allam , Sridhar R.; Chien, Yin-Hsiu; Giugliani, Roberto; Kallish, Staci; Kineen, Sabina; Lidove, Olivier; Niu, Dauming; Olivotto, Iacopo; Politei, Juan Manuel; Rakoski, Paul; Torra, Roser; Tondel, Camilla; Hughes, Derralynn A. (2023) [Artículo de periódico]
      Objective: Fabry 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 ...