FDA approved a lifesaving therapy for children with a rare genetic disease. It might become the world’s most expensive drug
A New Lease of Life: FDA Approves Expensive Gene Therapy for Rare and Devastating Condition, Metachromatic Leukodystrophy The United States Food and Drug Administration (FDA) has made a groundbreaking decision by approving the first-ever therapy for Metachromatic Leukodystrophy (MLD), a rare and devastating condition that typically claims the lives of affected children before they reach…
