FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with...
In recognition of World Rare Disease Day 2010, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) announced that they will collaborate together and now accept a single...
View ArticleMoms Receive EU Orphan Drug Designation From European Medicines Agency For...
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...
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