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

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 chaperone therapy migalastat to the available treatment options for Fabry disease...

Descripción completa

Detalles Bibliográficos
Autores principales: Bichet, Daniel G., Hopkin, Robert J., Aguiar, Patrício, Allam, Sridhar R., Chien, Yin-Hsiu, Giugliani, Roberto, Kallish, Staci, Kineen, Sabina, Lidove, Olivier, Niu, Dau-Ming, Olivotto, Iacopo, Politei, Juan, Rakoski, Paul, Torra, Roser, Tøndel, Camilla, Hughes, Derralynn A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Medicine
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10505750/
https://www.ncbi.nlm.nih.gov/pubmed/37727761
http://dx.doi.org/10.3389/fmed.2023.1220637

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10505750/
https://www.ncbi.nlm.nih.gov/pubmed/37727761
http://dx.doi.org/10.3389/fmed.2023.1220637

Ejemplares similares