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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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