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Horizon Gift Establishes First Clinical Team Endowment at CNRDI

Monday, February 26, 2018

Children’s National and Horizon Pharma plc announced today the creation of the Horizon Pharma Clinical Care Endowment, the first clinical team endowment at the Children’s National Rare Disease Institute (CNRDI). The endowment is made possible by a generous six-year, $3 million commitment from Horizon Pharma USA, Inc., a wholly owned subsidiary of Horizon Pharma plc – a biopharmaceutical company dedicated to improving the lives of people living with rare diseases.

Currently, it is estimated that one in 10 Americans has a rare disease – approximately 80 percent of which are genetically based. Additionally, the National Institutes of Health (NIH) reports that more than 80 percent are childhood diseases and more than 25 percent of children admitted to pediatric hospitals have a rare disease.

The Horizon Pharma Clinical Care Endowment will provide stable support for an expert care team at the CNRDI. Each team will consist of a clinical geneticist and support team members – such as genetic counselors, nutritionists, and social workers – all specializing in the care of children with rare disease.

“Patients and families with rare conditions deserve a medical home – a trusted place with the medical knowledge to provide quick, clear answers and the expert care they need,” says Marshall Summar, MD, director of the CNRDI.  “We are grateful for Horizon and their support of our mission to make the Children’s National Rare Disease Institute that home. This endowment will support a dedicated team that can provide optimal, comprehensive care to more patients and ensure that families have a trusted source for all aspects of their health care.”

The long-term support provided by the Horizon Pharma Clinical Care Endowment will give the CNRDI a firm foundation for treating patients earlier, more consistently, and over the course of their lifetimes. Horizon’s commitment marks the first donor-funded endowment at the CNRDI.

“The Children’s National Rare Disease Institute is an important step in better defining the journey from diagnosis to treatment for people living with rare disease and their families,” said Timothy P. Walbert, chairman, president and chief executive officer, Horizon Pharma plc. “We consistently hear stories from people who spent many years in search of a diagnosis, followed by more years searching for the right team of specialists – suffering through a painful journey with devastating consequences. Our hope is that the Horizon Pharma Clinical Care Endowment and the Institute will disrupt this all-too-familiar rare disease trajectory by joining exceptional rare disease specialists together in one place.”

The CNRDI is a first-of-its-kind center focused exclusively on advancing the care and treatment of children and adults with rare genetic diseases. It is the first National Organization for Rare Disorders (NORD) Center of Excellence and aims to provide a medical home for patients and families seeking the most advanced care and expertise for rare genetic conditions that remain largely unknown to the general medical community.

This commitment is the second major commitment to CNRDI already this year. In January, biopharmaceutical company Retrophin committed $3 million over six year to create the Retrophin Rare Disease Network at Children's National.

Philanthropic support is critical to furthering our understanding of rare diseases and pursuing new therapies and cures. To learn how you can support this important work, please contact Miriam Selman at mselman@childrensnational.org or 301-565-8440.


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