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Puncturing the Orphan Drug Bubble

This article was originally published in RPM Report

Executive Summary

BioMarin’s investor day highlighted a company in transformation: on the brink of profitability based on a stable of rare disease therapies, and generating buzz for an early stage research compound in oncology. Balancing those two portfolios may be challenging – and framing appeals to investors may be counterproductive in protecting the orphan drug model.

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