New research suggests that enzyme replacement therapy can make a major difference in the lives of people with Fabry disease, a rare illness that affects the kidneys, heart and brain.
The progressive disease, linked to the lack of an enzyme that helps metabolic pathways function in the body, can be fatal because it boosts the risk for a stroke, heart attack and kidney disease.
A study published Dec. 1 in The Lancet analyzed a treatment that replaced an enzyme called agalsidase alfa.
The researchers found that the treatment reduced signs of heart disease in people who had oversized hearts. It also appeared to improve kidney function, lower pain levels and improve scores on quality-of-life surveys.
"Enzyme-replacement therapy has only been available typically since 2001, and, thus, duration of exposure might be too short to assess whether this strategy prolongs life," the researchers wrote in an accompanying commentary. Whether the treatment, which they described as costly, helps people live longer needs confirmation, they noted.