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Therapy Slows Kidney Function Decline in Fabry Disease

Enzyme replacement with agalsidase alfa stabilizes kidney function

FRIDAY, April 10 (HealthDay News) -- Enzyme replacement therapy with agalsidase alfa in patients with Fabry disease, an X-linked disorder of glycosphingolipid metabolism, slows the rate of decline of kidney function, according to research published online April 8 in the Journal of the American Society of Nephrology.

Michael L. West, M.D., from Dalhousie University in Halifax, Nova Scotia, Canada, and colleagues analyzed data on kidney function in 108 male patients with Fabry disease who took part in randomized clinical trials of placebo versus enzyme replacement therapy with agalsidase alfa.

The researchers found that among non-hyperfiltrating patients, treatment with agalsidase alfa slowed the rate of decline in the glomerular filtration rate (GFR), with a mean annualized rate of change of -7.0 mL/min per 1.73 m2 for placebo compared with -2.9 mL/min per 1.73 m2 for agalsidase alfa. There was no change in proteinuria after agalsidase alfa treatment. After adjusting for relevant variables, GFR and proteinuria level at baseline were significant predictors of the rate of decline of GFR, the authors report.

"This summary represents the largest group of male patients who had Fabry disease and for whom the effects of enzyme replacement therapy on kidney function have been studied," West and colleagues conclude. "These data suggest that agalsidase alfa may stabilize kidney function in these patients."

Shire Human Genetic Therapies provided editorial assistance, statistical analysis, and reviewed the final manuscript. Several authors reported financial relationships with Shire and other pharmaceutical and biotechnology companies.

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