Thelansis

  Leukocyte adhesion deficiency type II (LAD-II) is a form of LAD (see this term) characterized by recurrent bacterial infections, severe growth delay, and severe intellectual deficit.   Etiology- LAD-II is a carbohydrate-deficient glycoprotein syndrome (CDG syndrome) and is therefore also referred to as CDG IIc. It results from mutations in the SLC35C1 gene (11p11.2), encoding the guanosine 5'-diphosphate (GDP)-fucose transporter localized in the Golgi apparatus. This is a specific fucose transporter that translocates GDP-fucose from the cytosol to the Golgi where it is used as a substrate for fucosylation.   Epidemiology- LAD-II is extremely rare: less than 10 cases have been reported so far   The competitive landscape of Leukocyte Adhesion Deficiency Type II 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...