Congratulations to Sharon Terry, Jouni Uitto, and All Rare Disease Heroes!


January 30, 2013

This article first appeared in the January 2013 PXE International eNewsletter

Jana Monaco and Sharon Terry, both parents of two children with genetic conditions, receive honors at the January 7, 2013
FDA Heroes Celebration.

On January 7, 2013 the U.S. Food and Drug Administration (FDA)celebrated the 30th anniversary of the passing of the Orphan Drug Act. During the celebration, Sharon Terry, CEO of PXE International and President and CEO of Genetic Alliance, was recognized by the FDA as one of 30 rare disease heroes for advocacy for innovative and collaborative approaches for rare disease products. PXE researcher Jouni Uitto of Jefferson Medical College was also recognized for basic research in rare diseases. The FDA commemorated more than 30 years of dedicated service by all stakeholders in the rare disease community, including the patient advocates, legislators, the research community, clinicians and industry.

The passage of the Orphan Drug Act on January 4, 1983 was important because it created – for the first time – incentives to develop desperately needed medical products for Americans suffering with rare diseases. Since its passage, over 2700 products in development have been designated as orphan drugs through the Orphan Drug Designation Program and over $290 million has been awarded to clinical studies through the Orphan Products Grants Program.