Thelansis

  A rare disorder of renal tubular amino acid transport characterized by the recurrent formation of kidney cystine stones.   Etiology- Cystinuria is inherited in an autosomal recessive pattern.  Cystinuria is caused by changes (mutations) in the SLC3A1 and SLC7A9 genes. These genes encode a protein complex that helps control the reabsorption of amino acids (such as cystine) in the kidneys. Mutations in these genes disrupt the function of the protein complex, causing cystine to become more concentrated in the urine.   Epidemiology- The prevalence of cystinuria has high ethnographic variation, ranging from 1:2,500 in the Libyan Jewish population to 1:100,000 in Sweden. The mean global value is estimated at 1:7,000.   The competitive landscape of Cystinuria 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 i...