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.Download