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Rare Disease: An Integrated, Patient-centered Approach to Research and Commercialization

September 29, 2016

Although rare diseases individually affect very few patients, the collective impact is staggering. Globally, the list of rare diseases has grown to approximately 7,000, and 350 million people are living with a rare disease. These diseases are serious, often life-threatening, and approximately half of those affected are children. The lack of available treatments leaves patients and their families searching for new options and new hope. Regulatory incentives such as the orphan products designation and the availability of Pediatric Rare Disease Priority Review Vouchers have increased the interest in rare disease research.

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