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

Long-Term Effect of Antibodies against Infused Alpha-Galactosidase A in Fabry Disease on Plasma and Urinary (lyso)Gb3 Reduction and Treatment Outcome

(2012) Saskia M. Rombach et al.   PLoS One

Inappropriately high left ventricular mass in patients with type 2 diabetes mellitus and no overt cardiac disease. The DYDA study

(2011) Giovanni Cioffi et al.   JOURNAL OF HYPERTENSION

Cardiovascular Events in Patients With Fabry Disease

(2011) Manesh R. Patel et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Response of women with Fabry disease to enzyme replacement therapy: Comparison with men, using data from FOS—the Fabry Outcome Survey

(2011) Derralynn A. Hughes et al.   MOLECULAR GENETICS AND METABOLISM

Renal outcomes of agalsidase beta treatment for Fabry disease: role of proteinuria and timing of treatment initiation

(2011) D. G. Warnock et al.   NEPHROLOGY DIALYSIS TRANSPLANTATION

Limitations of drug registries to evaluate orphan medicinal products for the treatment of lysosomal storage disorders

(2011) Carla EM Hollak et al.   Orphanet Journal of Rare Diseases

Plasma globotriaosylsphingosine: Diagnostic value and relation to clinical manifestations of Fabry disease

(2010) S.M. Rombach et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

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

Long-Term Effects of Enzyme Replacement Therapy on Fabry Cardiomyopathy

(2009) Frank Weidemann et al.   CIRCULATION

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

Baseline characteristics of patients enrolled in the Canadian Fabry Disease Initiative

(2009) S. Sirrs et al.   MOLECULAR GENETICS AND METABOLISM

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

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

Elevated globotriaosylsphingosine is a hallmark of Fabry disease

(2008) J. M. Aerts et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA