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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...
Autores principales: | Narita, Ichiei, Ohashi, Toya, Sakai, Norio, Hamazaki, Takashi, Skuban, Nina, Castelli, Jeffrey P., Lagast, Hjalmar, Barth, Jay A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer Singapore
2019
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Materias: | |
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 |
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