Dual therapy with migalastat and agalsidase-beta in a patient with Fabry disease with progressing hypertrophic cardiomyopathy

Chanan Stauffer; Manisha Balwani; Robert J. Desnick; Luca Fierro; Jaya Ganesh

doi:10.1016/j.ymgme.2020.12.250

Dual therapy with migalastat and agalsidase-beta in a patient with Fabry disease with progressing hypertrophic cardiomyopathy

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.250 ◽

2021 ◽

Vol 132 (2) ◽

pp. S103

Author(s):

Chanan Stauffer ◽

Manisha Balwani ◽

Robert J. Desnick ◽

Luca Fierro ◽

Jaya Ganesh

Keyword(s):

Hypertrophic Cardiomyopathy ◽

Fabry Disease ◽

Dual Therapy ◽

Agalsidase Beta

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Human medicines European public assessment report (EPAR): Fabrazyme, agalsidase beta, Fabry Disease, Date of authorisation: 03/08/2001, Revision: 26, Status: Authorised

Case Medical Research ◽

10.31525/cmr-19715f1 ◽

2019 ◽

Author(s):

Keyword(s):

Fabry Disease ◽

Agalsidase Beta ◽

Assessment Report ◽

European Public ◽

European Public Assessment Report

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Quantitative comparison of 2D and 3D late gadolinium enhancement MR imaging in patients with Fabry disease and hypertrophic cardiomyopathy

International Journal of Cardiology ◽

10.1016/j.ijcard.2016.04.175 ◽

2016 ◽

Vol 217 ◽

pp. 167-173 ◽

Cited By ~ 8

Author(s):

F. Morsbach ◽

S. Gordic ◽

C. Gruner ◽

M. Niemann ◽

R. Goetti ◽

...

Keyword(s):

Late Gadolinium Enhancement ◽

Hypertrophic Cardiomyopathy ◽

Mr Imaging ◽

Fabry Disease ◽

Quantitative Comparison ◽

Gadolinium Enhancement ◽

2D And 3D

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Agalsidase alfa and agalsidase beta in the treatment of Fabry disease: does the dose really matter?

Genetics in Medicine ◽

10.1038/gim.2014.79 ◽

2014 ◽

Vol 17 (1) ◽

pp. 21-23 ◽

Cited By ~ 3

Author(s):

Antonio Pisani ◽

Eleonora Riccio ◽

Massimo Sabbatini

Keyword(s):

Fabry Disease ◽

Agalsidase Beta ◽

Agalsidase Alfa

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66 Prevalence of fabry disease among patients diagnosed hypertrophic cardiomyopathy and unexplained left ventricular hypertrophy in ST. James Hospital (2013–2015)

10.1136/heartjnl-2016-310523.66 ◽

2016 ◽

Author(s):

H Sulaiman ◽

E Finegan ◽

A Rakovac Tisdall ◽

R Murphy

Keyword(s):

Hypertrophic Cardiomyopathy ◽

Left Ventricular Hypertrophy ◽

Fabry Disease ◽

Ventricular Hypertrophy ◽

Left Ventricular

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Female Fabry disease: significant improvement of Fabry disease-related gastrointestinal symptoms in a large cohort of female patients treated with agalsidase beta: data from the Fabry Registry

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2013.12.286 ◽

2014 ◽

Vol 111 (2) ◽

pp. S114-S115

Author(s):

William Wilcox ◽

Ulla Feldt-Rasmussen ◽

Ana Maria Martins ◽

Alberto Ortiz ◽

Frank Weidemann ◽

...

Keyword(s):

Fabry Disease ◽

Gastrointestinal Symptoms ◽

Large Cohort ◽

Agalsidase Beta ◽

Female Patients

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Value of Electrocardiogram in the Differentiation of Hypertensive Heart Disease, Hypertrophic Cardiomyopathy, Aortic Stenosis, Amyloidosis, and Fabry Disease

The American Journal of Cardiology ◽

10.1016/j.amjcard.2011.09.052 ◽

2012 ◽

Vol 109 (4) ◽

pp. 587-593 ◽

Cited By ~ 14

Author(s):

Mehdi Namdar ◽

Jan Steffel ◽

Sandra Jetzer ◽

Christian Schmied ◽

David Hürlimann ◽

...

Keyword(s):

Heart Disease ◽

Hypertrophic Cardiomyopathy ◽

Aortic Stenosis ◽

Fabry Disease ◽

Hypertensive Heart Disease

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Use of Myocardial T1 Mapping at 3.0 T to Differentiate Anderson-Fabry Disease from Hypertrophic Cardiomyopathy

Radiology ◽

10.1148/radiol.2018172613 ◽

2018 ◽

Vol 288 (2) ◽

pp. 398-406 ◽

Cited By ~ 24

Author(s):

Gauri R. Karur ◽

Sean Robison ◽

Robert M. Iwanochko ◽

Chantal F. Morel ◽

Andrew M. Crean ◽

...

Keyword(s):

Hypertrophic Cardiomyopathy ◽

Fabry Disease ◽

T1 Mapping ◽

Myocardial T1 ◽

3.0 T

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Improvement of Fabry Disease-Related Gastrointestinal Symptoms in a Significant Proportion of Female Patients Treated with Agalsidase Beta: Data from the Fabry Registry

JIMD Reports - JIMD Reports, Volume 38 ◽

10.1007/8904_2017_28 ◽

2017 ◽

pp. 45-51 ◽

Cited By ~ 9

Author(s):

William R. Wilcox ◽

Ulla Feldt-Rasmussen ◽

Ana Maria Martins ◽

Alberto Ortiz ◽

Roberta M. Lemay ◽

...

Keyword(s):

Fabry Disease ◽

Significant Proportion ◽

Gastrointestinal Symptoms ◽

Agalsidase Beta ◽

Female Patients

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Successful Management of Enzyme Replacement Therapy in Related Fabry Disease Patients with Severe Adverse Events by Switching from Agalsidase Beta (Fabrazyme®) to Agalsidase Alfa (Replagal®)

10.1007/8904_2014_304 ◽

2014 ◽

Cited By ~ 1

Author(s):

Kazuya Tsuboi ◽

Hiroshi Yamamoto ◽

Fuji Somura ◽

Hiromi Goto

Keyword(s):

Adverse Events ◽

Enzyme Replacement Therapy ◽

Fabry Disease ◽

Replacement Therapy ◽

Agalsidase Beta ◽

Successful Management ◽

Enzyme Replacement ◽

Agalsidase Alfa ◽

Severe Adverse Events

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Efficacy and safety of JR-051, a biosimilar of agalsidase beta, in patients with Fabry disease; results of a multicenter, open-label phase 3 study in Japan

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2017.12.201 ◽

2018 ◽

Vol 123 (2) ◽

pp. S79

Author(s):

Minako Kobayashi ◽

Tatsuyoshi Yamamoto ◽

Mariko Yamaoka ◽

Teiji Tomio

Keyword(s):

Fabry Disease ◽

Agalsidase Beta ◽

Efficacy And Safety ◽

Open Label ◽

Phase 3 ◽

Open Label Phase

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