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BioMarin Highlights Advances In Orphan Drug Pipeline

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

BioMarin highlighted research in several rare disease areas at an Oct. 19 R&D day, its first in over two years. Increasingly, drug development for rare disorders such as Gaucher or Pompe disease is an attractive area for even the biggest pharmaceutical players, in part because of the potentially lower regulatory and reimbursement hurdles associated with developing therapies to treat disorders where there is such great unmet medical need.

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