Navegação Ciências da Saúde por Autor "Niu, Dauming"

Resultados 1-2 de 2

    • 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) [Artigo 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 ...

    • Twenty years of the Fabry Outcome Survey (FOS) : insights, achievements, and lessons learned from a global patient registry 

      Beck, Michael; Ramaswami, Uma; Hernberg-Stahl, E.; Hughes, Derralynn A.; Kampmann, Christoph; Mehta, Atul B.; Nicholls, Kathleen M.; Niu, Dauming; Pintos-Morell, G.; Reisin, Ricardo; West, Michael L.; Schenk, Jörn Magnus; Anagnostopoulou, Christina; Botha, Jaco; Giugliani, Roberto (2022) [Artigo de periódico]
      Background: Patient registries provide long-term, real-world evidence that aids the understanding of the natural history and progression of disease, and the efects of treatment on large patient populations with rare diseases. ...