Thelansis

  Scleroderma is the common name for a broad class of rare, fatal, inherited disorders of the nervous system also known as neuronal ceroid lipofuscinoses, or NCLs. Etiology- Scleroderma is an inherited genetic disorder that appears to affect the function of tiny bodies within cells called lysosomes. In Scleroderma/NCLs, the mutated genes do not produce the proper amounts of proteins important for lysosomal function. Epidemiology- Scleroderma and other forms of NCL are relatively rare, occurring in an estimated 2 to 4 of every 100,000 births in the United States. The competitive landscape of Scleroderma includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary. KOLs insights of Scleroderma across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscap...