Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting | Zendy

Raphael Schiffmann | Zendy; Markus Ries | Zendy; Margaret Timmons | Zendy; John T. Flaherty | Zendy; Roscoe O. Brady | Zendy

Open Access

Long-term therapy with agalsidase alfa for Fabry disease: safety and effects on renal function in a home infusion setting

Author(s) -

Raphael Schiffmann,

Markus Ries,

Margaret Timmons,

John T. Flaherty,

Roscoe O. Brady

Publication year - 2005

Publication title -

nephrology dialysis transplantation

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.654

H-Index - 168

eISSN - 1460-2385

pISSN - 0931-0509

DOI - 10.1093/ndt/gfi152

Subject(s) - enzyme replacement therapy , medicine , fabry disease , renal function , globotriaosylceramide , renal replacement therapy , kidney disease , urology , alpha galactosidase , gastroenterology , kidney , endocrinology , disease

Fabry disease is an X-linked disorder of glycosphingolipid catabolism that is the result of an intracellular deficiency in the lysosomal enzyme alpha-galactosidase A (alpha-Gal A). This enzymatic defect results in the accumulation of globotriaosylceramide (Gb(3)) within cells and causes progressive neurological, cardiovascular and renal dysfunction. Our objective is to describe the safety and renal effects of long-term enzyme replacement therapy.

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