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

Molecular Genetics and Metabolism - Tập 131 - Trang 219-228 - 2020
Ulla Feldt-Rasmussen1, Derralynn Hughes2, Gere Sunder-Plassmann3, Suma Shankar4, Khan Nedd5, Iacopo Olivotto6, Damara Ortiz7, Toya Ohashi8, Takashi Hamazaki9, Nina Skuban10, Julie Yu10, Jay A. Barth10, Kathleen Nicholls11
1Department of Medical Endocrinology and Metabolism, Rigshospitalet, Copenhagen University Hospital, Copenhagen, Denmark
2Department of Haematology, Royal Free London NHS Foundation Trust and University College London, London, UK
3Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna, Vienna, Austria
4Department of Pediatrics, University of California-Davis, Davis, CA, USA
5Infusion Associates, Grand Rapids, MI, USA
6Cardiomyopathy Unit, Careggi University Hospital, Florence, Italy
7Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center, Pittsburgh, PA, USA
8Jikei University Hospital, Tokyo, Japan
9Department of Pediatrics, Osaka City University Graduate School of Medicine, Osaka, Japan
10Amicus Therapeutics, Inc, Cranbury, NJ, USA.
11Department of Nephrology, Royal Melbourne Hospital, University of Melbourne, Parkville, VIC, Australia

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Molecular Genetics and Metabolism

Tập 131

219-228

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