Pharma Investors Look for Lessons After BioMarin FDA Defeat

  • Sarepta's eteplirsen gives investors different leap of faith
  • DMD's unmet need: Without treatment, `the boys all die'

The rejection of a BioMarin Pharmaceutical Inc. medication by U.S. regulators left patients and investors questioning how drugmakers could improve their chances for approval of treatments for diseases that afflict tiny segments of the population.

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