WASHINGTON — Children’s National Health System and Horizon Pharma plc are pleased to announce 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 $3 million commitment over six years 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.
According to the NIH, any disease, illness or disorder that affects fewer than 200,000 people in the United States is considered to be rare. Yet, of the more than 7,000 rare diseases known to exist, fewer than 500 (roughly five percent) have a known treatment approved by the U.S. Food and Drug Administration (FDA).
Horizon Pharma plc is focused on researching, developing and commercializing innovative medicines that address unmet treatment needs for rare and rheumatic diseases. By fostering a growing pipeline of medicines in development and exploring all potential uses for currently marketed medicines, we strive to make a powerful difference for patients, their caregivers and physicians. For us, it’s personal: by living up to our own potential, we are helping others live up to theirs. For more information, please visit www.horizonpharma.com, follow us @HZNPplc on Twitter or like us on Facebook.
Media Contact: Gray Turner | 301-565-8523