Agalsidease-beta therapy for Fabry disease slowed progression of kidney, heart and nervous system complications and death compared to placebo, a new study found.
Fabry disease is a hereditary disease in which a faulty gene causes inadequate metabolism of certain fats in the body.
The randomized, double-blind, placebo-controlled trial was completed by only 74 people with advanced Fabry disease and mild to moderate kidney disease.
Only one third of participants developed clinical events. An editorial writer notes the high cost of lifelong therapy with agalasidase beta and says that therapy should be supplemented with the best standard medical care with particular emphasis on preventing stroke.