FDA Greenlights Groundbreaking Gene Therapy for Children Battling Rare Genetic Disease
In a landmark decision, the U.S. Food and Drug Administration (FDA) has given the green light to Lenmeldy (atidarsagene autotemcel), marking a significant breakthrough as the inaugural FDA-sanctioned gene therapy for children grappling with metachromatic leukodystrophy (MLD). MLD is a rare genetic disorder that makes people unable to function properly. It affects the brain and […]
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