Response of patients with Fabry disease with the amenable GLA mutation p.N215S to treatment with migalastat (original) (raw)
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study
Hjalmar Lagast
Clinical and Experimental Nephrology, 2019
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Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
Robert J Hopkin
Journal of Medical Genetics
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Oral Chaperone Therapy Migalastat for the Treatment of Fabry Disease: Potentials and Pitfalls of Real‐World Data
Einar Svarstad
Clinical Pharmacology & Therapeutics, 2019
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Treatment of Fabry Disease management with migalastat—outcome from a prospective 24 months observational multicenter study (FAMOUS)
Nesrin Karabul
European Heart Journal - Cardiovascular Pharmacotherapy
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Migalastat HCl Reduces Globotriaosylsphingosine (Lyso-Gb3) in Fabry Transgenic Mice and in the Plasma of Fabry Patients
Sheela Sitaraman, Kenneth Valenzano
PLoS ONE, 2013
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The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
William Wilcox
Genetics in medicine : official journal of the American College of Medical Genetics, 2016
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Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
Ozlem Goker-alpan
Journal of medical genetics, 2016
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Cardiovascular manifestations of Fabry disease: relationships between left ventricular hypertrophy, disease severity, and -galactosidase A activity
Katherine Sims
European Heart Journal, 2010
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Twenty-four-month α-galactosidase A replacement therapy in Fabry disease has only minimal effects on symptoms and cardiovascular parameters
Juha Koskenvuo
Journal of Inherited Metabolic Disease, 2008
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Effect of agalsidase alfa replacement therapy on fabry disease—related hypertrophic cardiomyopathy: A 12- to 36-month, retrospective, blinded echocardiographic pooled analysis
Christoph Kampmann
Clinical Therapeutics, 2009
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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase
Mathews Oneya
PloS one, 2015
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Effects of enzyme replacement therapy in adult patients with Fabry disease on cardiac structure and function: a retrospective cohort study of the Fabry Munster Study (FaMuS) data
Markus Engelen
BMJ open, 2012
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Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry
Gustavo Cabrera
Genetics in Medicine, 2013
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Progression of Fabry cardiomyopathy despite enzyme replacement therapy
Maurizio Pieroni, Deborah Cosmi
Circulation, 2013
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Late-Onset Cardiac Variant of Fabry Disease Responsive to Short-Term Treatment with Agalsidase Alpha
Walter Serra
Journal of Clinical & Experimental Cardiology, 2010
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Cardiomyopathy and kidney function in agalsidase beta‐treated female Fabry patients: a pre‐treatment vs. post‐treatment analysis
Bojan Vujkovac
ESC Heart Failure, 2020
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Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?
Sebastián Jaurretche
Healthcare
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Migalastat Tissue Distribution: Extrapolation From Mice to Humans Using Pharmacokinetic Modeling and Comparison With Agalsidase Beta Tissue Distribution in Mice
Anibh Das
Clinical Pharmacology in Drug Development, 2021
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Improvement of Cardiac Function During Enzyme Replacement Therapy in Patients With Fabry Disease: A Prospective Strain Rate Imaging Study
Frank Weidemann
Circulation, 2003
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Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy: Evidence for a Better Outcome With Early Treatment
Stefan Störk
Circulation, 2009
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Impact of Enzyme Replacement Therapy on Cardiac Morphology and Function and Late Enhancement in Fabry’s Cardiomyopathy
Frank Weidemann
American Journal of Cardiology, 2006
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Cardiac energy metabolism is disturbed in Fabry disease and improves with enzyme replacement therapy using recombinant human galactosidase A
Dietbert Hahn
European Journal of Heart Failure, 2011
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Cardio-renal outcomes with long-term agalsidase alfa enzyme replacement therapy: A 10-year Fabry Outcome Survey analysis
Jaco Botha
Molecular Genetics and Metabolism, 2016
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Paradoxical Response to Enzyme Replacement Therapy of Fabry Disease Cardiomyopathy
Andrea Frustaci
Circulation. Cardiovascular imaging, 2016
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<p>Cardio- Renal Outcomes With Long- Term Agalsidase Alfa Enzyme Replacement Therapy: A 10- Year Fabry Outcome Survey (FOS) Analysis</p>
Christoph Kampmann
Drug Design Development and Therapy, 2019
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Evaluation of the efficacy and safety of three dosing regimens of agalsidase alfa enzyme replacement therapy in adults with Fabry disease
Jacek Musial
Drug Design, Development and Therapy, 2015
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Downregulation of Mannose-6-Phosphate Receptors in Fabry Disease Cardiomyopathy: A Potential Target for Enzyme Therapy Enhancement
Margherita verardo
Journal of Clinical Medicine
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