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Focus on Rare Diseases and Orphan Drug Marketing

Taken together, rare diseases afflict as many as 30 million Americans - nearly 10% of the US population. To date, as many as 8,000 rare diseases have been identified but only 5% of rare diseases currently have any FDA-approved drug treatment.

Up to 80% of these disorders have a genetic origin; in fact, most are caused by defects in a single gene. Rare diseases take a large toll on children:

  • Approximately 50% of those affected by rare diseases are children
  • 30% of children with a rare disease will succumb to it before reaching 5 years of age
  • Rare diseases cause 35% of deaths occurring during the first 12 months of life

Read this whitepaper to find out the current state-of-play, the strategy to meet the demand, and the human resources aggressively treating patients with a rare disease.