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Committee for Orphan Medicinal Products | The Addi and Cassi Fund - Niemann Pick Type C

Friday, July 30, 2021

Moms Receive EU Orphan Drug Designation From European Medicines Agency For Novel Cholesterol Drug Called Cyclodextrin

September 14, 2011 by  
Filed under Featured Stories

Last year, after Dr. Caroline Hastings and I received the U.S. Food and Drug Administration orphan drug designation for Hydroxy-propyl-beta-cyclodextrin (HPBCD) for the treatment of Niemann Pick Type C disease, I was contacted by Sue French. Sue lives in the United Kingdom and her son William is also afflicted with the rare and fatal genetic […]