Thelansis

  An autosomal recessive lysosomal storage disease belongs to the oligosaccharidosis group (also called glycoproteins). Etiology- Mutations in the AGA gene cause aspartylglucosaminuria. The AGA gene provides instructions for producing an enzyme called aspartylglucosaminidase. Epidemiology- In Finland, where most cases are reported, there are an estimated 130 cases in 4.5 million persons. The condition is scarce in the rest of the world and affects persons of various heritages. The competitive landscape of Aspartylglucosaminuria (AGU) 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 Aspartylglucosaminuria (AGU) across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment parad...