Therapeutic goals in the treatment of Fabry disease

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

(2010) Olivier Lidove et al.   GENETICS IN MEDICINE

Four-Year Prospective Clinical Trial of Agalsidase Alfa in Children with Fabry Disease

(2010) Raphael Schiffmann et al.   JOURNAL OF PEDIATRICS

Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy

(2009) Frank Weidemann et al.   CIRCULATION

Effect of agalsidase alfa replacement therapy on fabry disease—related hypertrophic cardiomyopathy: A 12- to 36-month, retrospective, blinded echocardiographic pooled analysis

(2009) Christoph Kampmann et al.   CLINICAL THERAPEUTICS

Goal-oriented therapy with miglustat in Gaucher disease

(2009) Gregory M. Pastores et al.   CURRENT MEDICAL RESEARCH AND OPINION

Fabry disease in children and the effects of enzyme replacement treatment

(2009) Guillem Pintos-Morell et al.   EUROPEAN JOURNAL OF PEDIATRICS

Life expectancy and cause of death in males and females with Fabry disease: Findings from the Fabry Registry

(2009) Stephen Waldek et al.   GENETICS IN MEDICINE

A 4-year study of the efficacy and tolerability of enzyme replacement therapy with agalsidase alfa in 36 women with Fabry disease

(2009) Catharina Whybra et al.   GENETICS IN MEDICINE

Effects of enzyme-replacement therapy in patients with Anderson-Fabry disease: a prospective long-term cardiac magnetic resonance imaging study

(2009) M Imbriaco et al.   HEART

Cardiac challenges in patients with Fabry disease

(2009) F. Weidemann et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY

Can we use statins to prevent stroke in Fabry disease?

(2009) J. M. Politei   JOURNAL OF INHERITED METABOLIC DISEASE

Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey

(2009) A Mehta et al.   JOURNAL OF MEDICAL GENETICS

Guidelines to Diagnosis and Monitoring of Fabry Disease and Review of Treatment Experiences

(2009) Ana Maria Martins et al.   JOURNAL OF PEDIATRICS

Agalsidase Alfa and Kidney Dysfunction in Fabry Disease

(2009) M. West et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

New Equations to Estimate GFR in Children with CKD

(2009) G. J. Schwartz et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Enzyme replacement therapy with agalsidase alfa in patients with Fabry's disease: an analysis of registry data

(2009) A Mehta et al.   LANCET

A validated disease severity scoring system for Fabry disease

(2009) Edward H. Giannini et al.   MOLECULAR GENETICS AND METABOLISM

Reliability of quantitative sudomotor axon reflex testing and quantitative sensory testing in neuropathy of impaired glucose regulation

(2009) Amanda Peltier et al.   MUSCLE & NERVE

Monitoring renal function in children with Fabry disease: comparisons of measured and creatinine-based estimated glomerular filtration rate

(2009) C. Tondel et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

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

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

Fabry disease

(2009) Raphael Schiffmann   PHARMACOLOGY & THERAPEUTICS

Stroke in Fabry Disease Frequently Occurs Before Diagnosis and in the Absence of Other Clinical Events

(2009) Katherine Sims et al.   STROKE

Frequency of Unrecognized Fabry Disease Among Young European-American and African-American Men With First Ischemic Stroke

(2009) Marcella A. Wozniak et al.   STROKE

Onset and progression of the Anderson–Fabry disease related cardiomyopathy

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

Clinical usefulness of tissue Doppler imaging in predicting preclinical Fabry cardiomyopathy

(2008) Rocio Toro et al.   INTERNATIONAL JOURNAL OF CARDIOLOGY

Treatment of Fabry disease with different dosing regimens of agalsidase: Effects on antibody formation and GL-3

(2008) Anouk C. Vedder et al.   MOLECULAR GENETICS AND METABOLISM

Nephropathy in males and females with Fabry disease: cross-sectional description of patients before treatment with enzyme replacement therapy

(2008) A. Ortiz et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Characterization of Fabry Disease in 352 Pediatric Patients in the Fabry Registry

(2008) Robert J Hopkin et al.   PEDIATRIC RESEARCH

Heart Valve Involvement in Fabry Cardiomyopathy

(2008) Frank Weidemann et al.   ULTRASOUND IN MEDICINE AND BIOLOGY

Effects of enzyme replacement therapy on the cardiomyopathy of Anderson Fabry disease: a randomised, double-blind, placebo-controlled clinical trial of agalsidase alfa

(2007) D A Hughes et al.   HEART

Safety and Efficacy of Enzyme Replacement Therapy with Agalsidase Beta: An International, Open-label Study in Pediatric Patients with Fabry Disease

(2007) J. Edmond Wraith et al.   JOURNAL OF PEDIATRICS

Females with Fabry disease frequently have major organ involvement: Lessons from the Fabry Registry

(2007) William R. Wilcox et al.   MOLECULAR GENETICS AND METABOLISM