Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial

Migalastat: First Global Approval

(2016) Anthony Markham   DRUGS

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

Gastrointestinal involvement in Fabry disease. So important, yet often neglected

(2015) J. Politei et al.   CLINICAL GENETICS

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

Intrafamilial phenotypic variability in four families with Anderson-Fabry disease

(2013) M. Rigoldi et al.   CLINICAL GENETICS

Gastroenterological Complications of Anderson-Fabry Disease

(2013) Piotr Buda et al.   CURRENT PHARMACEUTICAL DESIGN

Anderson-Fabry Disease: A Multiorgan Disease

(2013) Antonino Tuttolomondo et al.   CURRENT PHARMACEUTICAL DESIGN

Neurological Complications of Anderson-Fabry Disease

(2013) Antonino Tuttolomondo et al.   CURRENT PHARMACEUTICAL DESIGN

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

Fabry disease

(2010) Dominique P Germain   Orphanet Journal of Rare Diseases

CONGENITAL MUSCULAR DYSTROPHY WITH DEFECTIVE  -DYSTROGLYCAN, CEREBELLAR HYPOPLASIA, AND EPILEPSY

(2009) S. Messina et al.   NEUROLOGY

Reliability and validity of the Gastrointestinal Symptom Rating Scale (GSRS) and Quality of Life in Reflux and Dyspepsia (QOLRAD) questionnaire in dyspepsia: A six-country study

(2008) Károly R Kulich et al.   Health and Quality of Life Outcomes