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Rare diseases — those afflicting fewer than 200,000 individuals in the United States — individually affect relatively small groups of patients. But 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, and roughly 250 new rare diseases are described annually.

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

Given the limited number of patients suffering from any one disease, rare diseases historically were not a common focus of...

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