Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice-Reference-Cited by-同舟云学术

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Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice

Published:2021-04-19 Issue:9 Volume:10 Page:1075-1088
ISSN:2160-763X
Container-title:Clinical Pharmacology in Drug Development
language:en
Short-container-title:Clinical Pharmacology in Drug Development

Author:

Wu Yi Shuan¹,Khanna Richie²,Schmith Virginia¹,Lun Yi²,Shen Jin‐Song²,Garcia Anadina²,Dungan Leo²,Perry Anthony²,Martin Lukas²,Tsai Pai‐Chi²,Hamler Rick²,Das Anibh M.³,Schiffmann Raphael⁴,Johnson Franklin K.²

Affiliation:

1. Nuventra Pharma Sciences Durham North Carolina USA

2. Amicus Therapeutics, Inc. Cranbury New Jersey USA

3. Clinic for Paediatric Nephrology Hepatology and Metabolic Disorders Hannover Medical School Hannover Germany

4. Baylor Scott and White Research Institute Dallas Texas USA

Publisher

Wiley

Subject

Pharmacology (medical),Pharmaceutical Science

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1002/cpdd.941

Reference41 articles.

1. Fabry disease

2. Contribution of inflammatory pathways to Fabry disease pathogenesis

3. Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey

4. Fabry disease revisited: Management and treatment recommendations for adult patients

5. Biotherapeutic target or sink: analysis of the macrophage mannose receptor tissue distribution in murine models of lysosomal storage diseases

Cited by 6 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Comparative study on incorporation of three recombinant human α-galactosidase A drugs (agalsidases) into cultured fibroblasts and organs/tissues of Fabry mice;Molecular Genetics and Metabolism Reports;2024-09

2. Clinical outcomes in patients switching from agalsidase beta to migalastat: A Fabry Registry analysis;Journal of Inherited Metabolic Disease;2024-07-04

3. Investigating Fabry disease - some lessons learned;Rare Disease and Orphan Drugs Journal;2024-01-23

4. Development and validation of a liquid chromatography–tandem mass spectrometry method for the quantification of the acetyl‐coenzyme A competitive p300/CBP catalytic inhibitor A‐485 in biological samples;Biomedical Chromatography;2023-12-26

5. Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes;Journal of Medical Genetics;2022-12-21

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