FDA Approves First Gene Therapy for rare genetic disease affecting brain and nervous system in kids
The U.S. Food and Drug Administration has approved first Gene Therapy for rare genetic disease affecting brain and nervous system in kids. Lenmeldy (atidarsagene autotemcel) is the first FDA-approved gene therapy indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). Metachromatic leukodystrophy is […]