Low Survival To Adulthood Key To Vimizim Pediatric Priority Voucher
This article was originally published in Pharmaceutical Approvals Monthly
Executive Summary
FDA says that Morquio A syndrome qualifies as a rare pediatric disease because most patients with a severe form of the disease will not survive into adulthood, paving the way for BioMarin to receive the first rare pediatric disease priority review voucher.
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Review Of Reviews: Drug Review Profiles 2014
Pharmaceutical Approvals Monthly’s Drug Review Profile stories from 2014.
Review Of Reviews: Drug Review Profiles 2014
Pharmaceutical Approvals Monthly’s Drug Review Profile stories from 2014.