- ►nih.gov WR Wilcox, M Banikazemi, N Guffon, S Waldek … - The American Journal of Human Genetics, 2004 - Elsevier Elsewhere, we reported the safety and efficacy results of a multicenter phase 3
trial of recombinant human α-galactosidase A (rh-αGalA) replacement in
patients with Fabry disease. All 58 patients who were enrolled in the 20-wk ... Cited by 201 - Related articles - BL Direct - All 12 versions
- ►annals.org M Banikazemi, J Bultas, S Waldek, WR Wilcox … - Annals of internal medicine, 2007 - Am Coll Physicians Background: Fabry disease ( -galactosidase A deficiency) is a rare, X-linked
lysosomal storage disorder that can cause early death from renal, cardiac, and
cerebrovascular involvement. ... Objective: To see whether agalsidase beta ... Cited by 134 - Related articles - BL Direct - All 6 versions
RJ Desnick - Journal of inherited metabolic disease, 2004 - Springer Summary: Although ¢rst suggested by de Duve in 1964, enzyme replacement therapy
(ERT) for lysosomal storage diseases did not become a reality until the early
1990s when its safety and e¡ectiveness were demonstrated in type 1 Gaucher ... Cited by 109 - Related articles - BL Direct - All 7 versions
- ►nih.gov M Spada, S Pagliardini, M Yasuda, T Tukel, … - The American Journal of Human Genetics, 2006 - Elsevier The classic phenotype of Fabry disease, X-linked α-galactosidase A (α-Gal A)
deficiency, has an estimated incidence of 1 in 50,000 males. The recent
recognition of later-onset variants suggested that this treatable lysosomal ... Cited by 103 - Related articles - BL Direct - All 8 versions
CM Eng, DP Germain, M Banikazemi, DG … - Genetics in Medicine, 2006 - journals.lww.com From the 1 Baylor College of Medicine, Houston, Texas; 2 Hôpital Européen
Georges Pompidou, Paris, France; 3 Mount Sinai School of Medicine, New York, New
York; 4 University of Alabama at Birmingham, Birmingham, Alabama; 5 ... Cited by 74 - Related articles - BL Direct - All 3 versions
F Breunig, F Weidemann, J Strotmann, A … - Kidney international, 2006 - nature.com Enzyme replacement therapy (ERT) with recombinant human -galactosidase A (rh
GalA) enhances microvascular globotriaosylceramide clearance and improves
clinical symptoms in patients with Fabry disease. We evaluated whether ... Cited by 67 - Related articles - BL Direct - All 4 versions
- ►asnjournals.org P Kotanko, R Kramar, D Devrnja, E Paschke, … - Journal of the American Society of Nephrology, 2004 - Am Soc Nephrol ABSTRACT. Anderson-Fabry disease is possibly underdiagnosed in patients with
end-stage renal disease. Nationwide screening was therefore undertaken for
Anderson-Fabry disease among dialysis patients in Austria. Screening for - ... Cited by 62 - Related articles - BL Direct - All 7 versions
WR Wilcox, JP Oliveira, RJ Hopkin, A Ortiz, … - Molecular genetics and metabolism, 2007 - Elsevier Fabry disease (FD) is an X-linked lysosomal storage disease caused by
alpha-galactosidase A deficiency. The Fabry Registry is a global clinical effort
to collect longitudinal data on FD. In the past, most “carrier” females ... Cited by 58 - Related articles - All 4 versions
RJ Ziegler, SM Lonning, D Armentano, C Li, … - Molecular Therapy, 2004 - Elsevier The successful application of gene therapy for the treatment of genetic diseases
such as Fabry is reliant on the development of vectors that are safe and that
facilitate sustained expression of therapeutic levels of the transgene ... Cited by 57 - Related articles - All 5 versions
CM Eng, J Fletcher, WR Wilcox, S Waldek, CR … - Journal of inherited metabolic disease, 2007 - Springer J Inherit Metab Dis (2007) 30:184–192 DOI 10.1007/s10545-007-0521-2 ... Fabry
disease: Baseline medical characteristics of a cohort of 1765 ... CM Eng · J.
Fletcher · WR Wilcox · S. Waldek · CR Scott · DO Sillence · F. Breunig ... Cited by 57 - Related articles - BL Direct - All 5 versions
