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

Enzyme replacement therapy for Anderson-Fabry disease: A complementary overview of a Cochrane publication through a linear regression and a pooled analysis of proportions from cohort studies

(2017) Regina El Dib 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

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

Serum-Mediated Inhibition of Enzyme Replacement Therapy in Fabry Disease

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

Recommendations for initiation and cessation of enzyme replacement therapy in patients with Fabry disease: the European Fabry Working Group consensus document

(2015) Marieke Biegstraaten et al.   Orphanet Journal of Rare Diseases

Multiplex Tandem Mass Spectrometry Analysis of Novel Plasma Lyso-Gb3-Related Analogues in Fabry Disease

(2014) Michel Boutin et al.   ANALYTICAL CHEMISTRY

Improvement in Gastrointestinal and Health-related Quality of Life Outcomes After Conversion From Mycophenolate Mofetil to Enteric-coated Mycophenolate Sodium in Liver Transplant Recipients

(2014) M. Sterneck et al.   TRANSPLANTATION PROCEEDINGS

Analysis of left ventricular mass in untreated men and in men treated with agalsidase-β: data from the Fabry Registry

(2013) Dominique P. Germain et al.   GENETICS IN MEDICINE

Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications

(2013) F. Weidemann et al.   JOURNAL OF INTERNAL MEDICINE

A Phase 2 study of migalastat hydrochloride in females with Fabry disease: Selection of population, safety and pharmacodynamic effects

(2013) R. Giugliani et al.   MOLECULAR GENETICS AND METABOLISM

Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain

(2013) Saskia M Rombach et al.   Orphanet Journal of Rare Diseases

Novel Quantitative Method to Evaluate Globotriaosylceramide Inclusions in Renal Peritubular Capillaries by Virtual Microscopy in Patients With Fabry Disease

(2012) Laura Barisoni et al.   ARCHIVES OF PATHOLOGY & LABORATORY MEDICINE

The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy

(2012) S. Feriozzi et al.   Clinical Journal of the American Society of Nephrology

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

(2012) Dominique P Germain et al.   Orphanet Journal of Rare Diseases

Prognostic Indicators of Renal Disease Progression in Adults with Fabry Disease: Natural History Data from the Fabry Registry

(2010) C. Wanner et al.   Clinical Journal of the American Society of Nephrology

Expert opinion on temporary treatment recommendations for Fabry disease during the shortage of enzyme replacement therapy (ERT)

(2010) Gabor E. Linthorst et al.   MOLECULAR GENETICS AND METABOLISM

Fabry disease

(2010) Dominique P Germain   Orphanet Journal of Rare Diseases

Fabry disease: a review of current management strategies

(2010) A. Mehta et al.   QJM-AN INTERNATIONAL JOURNAL OF MEDICINE

Newborn screening for Fabry disease in Taiwan reveals a high incidence of the later-onsetGLAmutation c.936+919G>A (IVS4+919G>A)

(2009) Wuh-Liang Hwu et al.   HUMAN MUTATION

Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy

(2009) R. Schiffmann et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Onset and progression of the Anderson–Fabry disease related cardiomyopathy

(2008) Christoph Kampmann et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY