Oral Chaperone Therapy Migalastat for Treating Fabry Disease: Enzymatic Response and Serum Biomarker Changes After 1 Year

Long‐term effects of migalastat therapy in clinical practice are currently unknown. We evaluated migalastat efficacy and biomarker changes in a prospective, single‐center study on 14 patients with Fabry disease (55 ± 14 years; 11 men). After 1 year of open‐label migalastat therapy, patients showed s...

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Detalhes bibliográficos
Publicado no:Clin Pharmacol Ther
Principais autores: Müntze, Jonas, Gensler, Daniel, Maniuc, Octavian, Liu, Dan, Cairns, Tereza, Oder, Daniel, Hu, Kai, Lorenz, Kristina, Frantz, Stefan, Wanner, Christoph, Nordbeck, Peter
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2019
Assuntos:
Research
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6590383/
https://ncbi.nlm.nih.gov/pubmed/30506669
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/cpt.1321
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