Gene Therapy for Metachromatic Leukodystrophy: A Breakthrough Backed by NEJM—The Critical Role of Timing

Gene Therapy Offers Hope for Children with Metachromatic Leukodystrophy: Long-term Study Shows Promising Results Metachromatic leukodystrophy (MLD) is a rare and devastating genetic neurodegenerative disorder that causes progressive deterioration of motor and cognitive functions in children. This fatal disease is characterized by the accumulation of sulfatides due to mutations in the ARSA gene, w