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Science & Innovation

Faster Progress Against Rare Disease: Cincinnati Children's Hospital and Shire

Cincinnati ChildrensWhen does rare become common? When the subject is rare disease. Any one of the 7,000 known rare diseases impacts a small group of people, but addressing their impact means helping one out of every 10, or 30 million people in the U.S. and another 30 million in the EU.

This collective mindset is the approach scientists at the Cincinnati Children’s Hospital Medical Center in Avondale, Ohio are taking with their rare disease program. The team wanted to accelerate the process that transforms their research into treatments that can be delivered to patients. Shire joined the effort in 2015, bringing complementary scientific, developmental and commercialization expertise to the broad, three-year research collaboration.

“We are very excited to partner with Shire in this capacity as we strive to change the outcome for children with rare and complex diseases,” said Margaret Hostetter, M.D., chief medical officer of the hospital and director of its research foundation. “Cincinnati Children’s full spectrum of research capabilities – basic, clinical and translational – coupled with Shire’s drug-development expertise makes for a very powerful partnership that we hope will have an impact on patients around the world.”

The team is organized around making that impact quickly. The collaboration focuses on programs with the potential to deliver a developmental therapeutic candidate on an accelerated timeline – less than three years from project initiation. This collaboration is an example of the power of partnership to smooth the path from insight to innovation to potential introduction of a new therapy. For Shire, this partnership, and others like it, shows how to foster new approaches to yield life-changing medications for patients who need them.


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