Cost-effectiveness of enzyme replacement therapy for Fabry disease

Pricing and reimbursement of orphan drugs: the need for more transparency

(2011) Steven Simoens   Orphanet Journal of Rare Diseases

Using a genetic, observational study as a strategy to estimate the potential cost-effectiveness of pharmacological CCR5 blockade in dialysis patients

(2011) Friso L.H. Muntinghe et al.   Pharmacogenetics and Genomics

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

Baseline characteristics of patients enrolled in the Canadian Fabry Disease Initiative

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

Angiotensin inhibition in renovascular disease: A population-based cohort study

(2008) Daniel G. Hackam et al.   AMERICAN HEART JOURNAL

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