scholarly journals A case of Fabry's disease initially diagnosed after the initiation of hemodialysis, which was successfully controlled by enzyme replacement therapy

2010 ◽  
Vol 43 (1) ◽  
pp. 99-103
Author(s):  
Teruko Nakamura ◽  
Yukari Yamashita ◽  
Yoshiyuki Tomiyoshi ◽  
Shuichi Rikitake ◽  
Tomoya Kishi ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Fabry’S Disease ◽  
Fabry's Disease ◽  
Enzyme Replacement

Enzyme replacement therapy for Fabry's disease

The Lancet ◽  
2010 ◽  
Vol 375 (9725) ◽  
pp. 1523 ◽  
Author(s):  
Stephen Waldek ◽  
Dominique P Germain ◽  
Christoph Wanner ◽  
David G Warnock
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Fabry’S Disease ◽  
Fabry's Disease ◽  
Enzyme Replacement

scholarly journals Enzyme Replacement Therapy in Patients with Fabry's Disease

2007 ◽  
Vol 35 (4) ◽  
pp. 574-581 ◽  
Author(s):  
K Tsuboi
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Vascular Endothelial Cells ◽  
Vascular Endothelial ◽  
Fabry’S Disease ◽  
Fabry's Disease ◽  
Factors Associated ◽  
Enzyme Replacement ◽  
Corneal Clouding ◽  
Ambulatory Patients

Fabry's disease, a disorder affecting the gene for the lysosomal enzyme α-galactosidase A (α-GAL A), can cause accumulation of globotriaosylceramide (GL-3) in the vascular endothelial cells. Symptoms include pain, angiokeratoma, corneal clouding, and damage to the heart and kidneys. Human recombinant α-GAL A for use as an enzyme replacement therapy was launched in Japan in April 2004. Eleven ambulatory patients with Fabry's disease were given replacement α-GAL A therapy. Three patients died due to factors associated with Fabry's disease. The enzyme replacement therapies in the remaining eight patients continued safely without any notable adverse events. The following were observed: a lowering of the plasma levels of GL-3 in seven cases, an improvement in the daily activities in six cases, and a reduction in corneal clouding in three cases. Although careful observation is necessary, these results suggest that replacement α-GAL A therapy may be a safe and effective treatment of Fabry's disease.

Enzyme replacement therapy with agalsidase β improves cardiac involvement in Fabry's disease

2004 ◽  
Vol 66 (2) ◽  
pp. 158-165 ◽  
Author(s):  
L Spinelli ◽  
A Pisani ◽  
M Sabbatini ◽  
M Petretta ◽  
MV Andreucci ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Cardiac Involvement ◽  
Fabry’S Disease ◽  
Fabry's Disease ◽  
Enzyme Replacement ◽  
Agalsidase Β

Renal histology before and after effective enzyme replacement therapy in a patient with classical Fabry’s disease

2009 ◽  
Vol 71 (05) ◽  
pp. 550-556 ◽  
Author(s):  
S. Hirashio ◽  
T. Taguchi ◽  
T. Naito ◽  
K. Maki ◽  
S. Ogata ◽  
...  
Keyword(s):  
Enzyme Replacement Therapy ◽  
Replacement Therapy ◽  
Fabry’S Disease ◽  
Fabry's Disease ◽  
Enzyme Replacement ◽  
Renal Histology ◽  
Before And After
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