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Agalsidase-Beta Effective for Fabry Disease

Drug lowers risk of clinical events in patients with advanced Fabry disease

TUESDAY, Dec. 19 (HealthDay News) -- Agalsidase-beta lowers the risk of clinical events in patients with advanced Fabry disease, a rare, X-linked lysosomal storage disorder that affects heart and kidney function, according to a study in the Jan. 16 issue of the Annals of Internal Medicine.

Robert J. Desnick, M.D., Ph.D., from Mount Sinai School of Medicine in New York City, and colleagues randomized 82 patients with Fabry disease and mild to moderate kidney disease to intravenous placebo (31 patients) or agalsidase-beta (1 mg/kg, 51 patients) every two weeks for a median of 18.5 months. Nearly 90 percent of the patients were men.

Forty-two percent of placebo patients compared with 27 percent of agalsidase-beta patients had a clinical event (renal, cardiac or cerebrovascular event, or death). In the 74 protocol-adherent patients, after adjusting for baseline proteinuria, agalsidase-beta significantly delayed the time to first clinical event (hazard ratio 0.39). This risk reduction was most pronounced in patients with a preserved baseline glomerular filtration rate of more than 55 mL/min per 1.73 square meters (hazard ratio 0.19). Most adverse events were infusion-associated reactions.

The study "provides a crucial path toward lowering the morbidity of Fabry disease," states the author of an accompanying editorial.

The study was funded by the National Center for Research Resources and the Genzyme Corporation.

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