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Exceling at Accelerating in Rare Disease Drug Development

By: Rajesh Krishna, PhD, FAAPS and Steve Sibley

According to the National Institutes of Health, more than 30 million people in the United States are affected by a rare disease. Defined in the Orphan Drug Act as any disease that affects less than 200,000 people in the United States, rare diseases total more than 7,000 conditions. This means that based on 2020 census records, nearly one in 10 Americans is living with a rare condition, making seemingly “rare” diseases more common than ever before… (cont.)