Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes

BACKGROUND: Fabry disease is a rare, multisystemic disorder caused by GLA gene variants that lead to alpha galactosidase A deficiency, resulting in accumulation of glycosphingolipids and cellular dysfunction. Fabry-associated clinical events (FACEs) cause significant morbidity and mortality, yet the...

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Detalles Bibliográficos
Autores principales: Hughes, Derralynn A, Bichet, Daniel G, Giugliani, Roberto, Hopkin, Robert J, Krusinska, Eva, Nicholls, Kathleen, Olivotto, Iacopo, Feldt-Rasmussen, Ulla, Sakai, Norio, Skuban, Nina, Sunder-Plassmann, Gere, Torra, Roser, Wilcox, William R
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2023
Materias:
Therapeutics
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10359570/
https://www.ncbi.nlm.nih.gov/pubmed/36543533
http://dx.doi.org/10.1136/jmg-2022-108669

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