Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study

BACKGROUND: Fabry disease is a progressive X-linked lysosomal disorder. In this subgroup analysis of the global phase III ATTRACT study, the efficacy and safety of oral migalastat, a pharmacologic chaperone, were investigated in Japanese patients with Fabry disease. METHODS: Patients were randomly a...

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Detalles Bibliográficos
Autores principales: Narita, Ichiei, Ohashi, Toya, Sakai, Norio, Hamazaki, Takashi, Skuban, Nina, Castelli, Jeffrey P., Lagast, Hjalmar, Barth, Jay A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Singapore 2019
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7007427/
https://www.ncbi.nlm.nih.gov/pubmed/31889231
http://dx.doi.org/10.1007/s10157-019-01810-w

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7007427/
https://www.ncbi.nlm.nih.gov/pubmed/31889231
http://dx.doi.org/10.1007/s10157-019-01810-w

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