Pediatric Neuron Disease Gets Its First Gene Therapy
The FDA approved atidarsagene autotemcel (arsa-cel; Lenmeldy), the first gene therapy to treat children with pre-symptomatic late infantile, pre-symptomatic early juvenile, or early symptomatic early juvenile metachromatic leukodystrophy (MLD), the agency announced on Monday. “This is the first FDA-approved treatment option for children who have this rare genetic disease,” Peter Marks, MD, PhD, director of […]