BioMarin Pharmaceutical Inc.'s Dwarfism Medicine Marks Growth of $400,000 Rare-Disease Drugs

David Blaustein waited seven years for the phone call that finally came in December 2010 from BioMarin Pharmaceutical Inc. (BMRN), a maker of medicines for rare diseases. Blaustein’s 8-year-old son has achondroplasia, a genetic condition that’s the most common form of dwarfism. A doctor turned investor, Blaustein spent years trying to find any company focused on the disease, which affects fewer than 30,000 people globally and for which there are no approved medicines. BioMarin, he learned, was about to announce it was at work on what could be the first one.

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