Switch from enzyme replacement therapy to oral chaperone migalastat for treating fabry disease: real-life data

Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

(2019) Dominique P. Germain et al.   GENETICS IN MEDICINE

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

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

Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference

(2017) Raphael Schiffmann et al.   KIDNEY INTERNATIONAL

Retrospective study of long-term outcomes of enzyme replacement therapy in Fabry disease: Analysis of prognostic factors

(2017) Maarten Arends et al.   PLoS One

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

Treatment of Fabry’s Disease with the Pharmacologic Chaperone Migalastat

(2016) Dominique P. Germain et al.   NEW ENGLAND JOURNAL OF MEDICINE

Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease

(2015) M. Lenders et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

A New Equation to Estimate Glomerular Filtration Rate

(2013) Andrew S. Levey et al.   ANNALS OF INTERNAL MEDICINE

Enzyme replacement therapy in patients with Fabry disease: State of the art and review of the literature

(2012) Antonio Pisani et al.   MOLECULAR GENETICS AND METABOLISM

Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders

(2011) Kenneth J. Valenzano et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Effect of intravenous flumazenil on oral midazolam pharmacokinetics and pharmacodynamics for use as a cytochrome P450 3A probe

(2011) J.D. Ma et al.   INTERNATIONAL JOURNAL OF CLINICAL PHARMACOLOGY AND THERAPEUTICS

Effects of enzyme replacement therapy in Fabry disease—A comprehensive review of the medical literature

(2010) Olivier Lidove et al.   GENETICS IN MEDICINE

Fabry disease

(2010) Dominique P Germain   Orphanet Journal of Rare Diseases

The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines

(2009) E. R. Benjamin et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

(2009) Richie Khanna et al.   MOLECULAR THERAPY