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BioMarin’s Orphan Drug Clinical Strategy Is Rooted In Natural History

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

BioMarin has emerged at the forefront of orphan drug development by grounding its clinical trials in natural history studies. The specialty firm’s recent investor day highlighted how clinical trials in ultra-orphan disorders are informed by efforts to quantify the clinical course of often poorly-understood conditions to determine appropriate endpoints and dosing strategies.

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