Parents for Orphan Drug Designation

摘要

Last year Hydroxy-propyl-beta-cyclodextrin (HPBCD) received the orphan drug designation from U.S. Food and Drug Administration for the treatment of Niemann Pick Type C (NPC) disease. The Cyclodextrin News regularly reported on the advance of the procedure till the designation in the U.S. was achieved and also on the efforts of the mother of the twins suffering in NPC disease, in this rare genetic disorder of cholesterol metabolism in the brain.