Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study-Reference-Cited by-同舟云学术

Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

Published:2020-09 Issue:1-2 Volume:131 Page:219-228
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Feldt-Rasmussen Ulla,Hughes Derralynn,Sunder-Plassmann Gere,Shankar Suma,Nedd Khan,Olivotto Iacopo,Ortiz Damara,Ohashi Toya,Hamazaki Takashi,Skuban Nina,Yu Julie,Barth Jay A.,Nicholls Kathleen

Funder

Amicus Therapeutics, Inc

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference51 articles.

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4. Prevalence of lysosomal storage disorders;Meikle;JAMA,1999

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