FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy

The ULF is shared to be able to share this press release from the Food & Drug Association:

“Today, the U.S. Food and Drug Administration approved Lenmeldy (atidarsagene autotemcel), 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).” Read more here.