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Ultra rare diseases | The Addi and Cassi Fund - Niemann Pick Type C

Wednesday, December 1, 2021

FDA Grants Orphan Drug Status For Cyclodextrin Compound To Treat Fatal Genetic Cholesterol Disease

May 16, 2010 by  
Filed under Featured Stories

Drum roll, please! Dr. Caroline Hastings at Children’s Hospital Oakland and Research Center received a call on Friday afternoon from the U.S. Food and Drug Administration regarding the orphan drug submission we made at the end of February. While we have not received the official letter by mail (probably next week), it appears our Orphan […]