World-First Fabry Disease Treated With Gene Therapy

Fabry disease is a rare, chronic illness that can damage major organs and shorten lives. they have a gene called GLA that does not function correctly. As a result, their bodies are unable to make the correct version of an enzyme that breaks down a fat leading to problems in the kidneys, heart and brain.

from Medindia Health News https://ift.tt/3bygs5q