Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?
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Title
Fabry Disease: Switch from Enzyme Replacement Therapy to Oral Chaperone Migalastat: What Do We Know Today?
Authors
Keywords
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Journal
Healthcare
Volume 11, Issue 4, Pages 449
Publisher
MDPI AG
Online
2023-02-06
DOI
10.3390/healthcare11040449
References
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Related references
Note: Only part of the references are listed.- Chaperone Therapy in Fabry Disease
- (2022) Frank Weidemann et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
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- Long-term multisystemic efficacy of migalastat on Fabry-associated clinical events, including renal, cardiac and cerebrovascular outcomes
- (2022) Derralynn A Hughes et al. JOURNAL OF MEDICAL GENETICS
- Treatment of Fabry Disease management with migalastat—outcome from a prospective 24 months observational multicenter study (FAMOUS)
- (2021) Malte Lenders et al. European Heart Journal-Cardiovascular Pharmacotherapy
- Long-term follow-up of renal function in patients treated with migalastat for Fabry disease
- (2021) Daniel G. Bichet et al. Molecular Genetics and Metabolism Reports
- Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data
- (2020) Eleonora Riccio et al. EUROPEAN JOURNAL OF HUMAN GENETICS
- 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
- (2020) Ulla Feldt-Rasmussen et al. MOLECULAR GENETICS AND METABOLISM
- In Vitro and In Vivo Amenability to Migalastat in Fabry Disease
- (2020) Malte Lenders et al. Molecular Therapy-Methods & Clinical Development
- Migalastat: A Review in Fabry Disease
- (2019) Emma H. McCafferty et al. DRUGS
- Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study
- (2019) Derralynn A. Hughes et al. AMERICAN JOURNAL OF MEDICAL GENETICS PART A
- Mutation-specific Fabry disease patient-derived cell model to evaluate the amenability to chaperone therapy
- (2019) Malte Lenders et al. JOURNAL OF MEDICAL GENETICS
- Female Fabry disease patients and X-chromosome inactivation
- (2018) Patrycja Juchniewicz et al. GENE
- Fabry disease revisited: Management and treatment recommendations for adult patients
- (2018) Alberto Ortiz et al. MOLECULAR GENETICS AND METABOLISM
- Oral Chaperone Therapy Migalastat for Treating Fabry Disease: Enzymatic Response and Serum Biomarker Changes After 1 Year
- (2018) Jonas Müntze et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- Newborn screening for lysosomal storage disorders by tandem mass spectrometry in North East Italy
- (2017) Alberto B. Burlina et al. JOURNAL OF INHERITED METABOLIC DISEASE
- The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat
- (2016) Elfrida R. Benjamin et al. GENETICS IN MEDICINE
- Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study
- (2016) Derralynn A Hughes et al. JOURNAL OF MEDICAL GENETICS
- Characterization of Classical and Nonclassical Fabry Disease: A Multicenter Study
- (2016) Maarten Arends et al. JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
- Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat
- (2016) Dominique P. Germain et al. NEW ENGLAND JOURNAL OF MEDICINE
- X-chromosome inactivation in female patients with Fabry disease
- (2015) L. Echevarria et al. CLINICAL GENETICS
- Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders
- (2015) Giancarlo Parenti et al. MOLECULAR THERAPY
- Newborn screening for lysosomal storage disorders
- (2015) Dietrich Matern et al. SEMINARS IN PERINATOLOGY
- Fabry disease
- (2010) Dominique P Germain Orphanet Journal of Rare Diseases
- Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics
- (2009) Giancarlo Parenti EMBO Molecular Medicine
- The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease
- (2009) Richie Khanna et al. MOLECULAR THERAPY
- Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy
- (2009) R. Schiffmann et al. NEPHROLOGY DIALYSIS TRANSPLANTATION
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