Thelansis

  A rare lysosomal disease characterized by accumulation of sulfatides in the central and peripheral nervous system due to deficiency of the enzyme arylsulfatase A, leading to demyelination. Etiology- This condition is inherited in an autosomal recessive pattern and is caused by mutations in the ARSA and PSAP genes. Epidemiology- The true prevalence rate of MLD is unknown but is estimated to be between 1 in 40,000 and 1 in 160,000. The Navajo also have a higher prevalence rate of 1 in every 2,500 people. In certain populations in the Middle East, these numbers may be even higher. The competitive landscape of Metachromatic Leukodystrophy includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary. KOLs insights of Metachromatic Leukodystrophy across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. In...