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Children's Hospital Participates in New NIH Study of Rare Genetic Airways Disorders

May 19, 2006

Children’s Hospital and Regional Medical Center is one of four institutions participating in a new study of rare genetic airways disorders, which is being launched as part of the National Institute of Health’s Rare Diseases Clinical Research Network (RDCRN).

Children’s Hospital and Regional Medical Center is one of four institutions participating in a new study of rare genetic airways disorders, which is being launched as part of the National Institute of Health’s Rare Diseases Clinical Research Network (RDCRN).

The research study will be lead by Dr. Margaret Rosenfeld, a researcher at Children’s Hospital and associate professor of pediatrics at the University of Washington School of Medicine (UWSOM), along with Dr. Ron Gibson, professor of pediatrics at UWSOM, and Dr. Moira Aitken, professor of medicine at UWSOM.

The study under way currently will attempt to find new, more effective ways of diagnosing rare genetic airways disorders such as primary ciliary dyskinesia. The network also plans to conduct a longitudinal study of the natural history of primary ciliary dyskinesia. Other institutions participating in the network, called the Genetic Diseases of Mucociliary Clearance Consortium, include the University of North Carolina at Chapel Hill, Washington University in St. Louis, and The Children’s Hospital in Denver.

“We feel privileged to be one of four participating sites in this national network,” said Rosenfeld. “The goal of this network is to standardize the approach to diagnosis and treatment of primary ciliary dyskinesia and other rare disorders of mucociliary clearance.”

The RDCRN expects to open more than 20 studies in the next few months at about 50 sites across the United States, the United Kingdom, Japan, and Brazil. The RDCRN has received five-year funding awards totaling $71 million and is coordinated primarily by two NIH components-the Office of Rare Diseases (ORD) and the National Center for Research Resources (NCRR). A rare disease is defined as a disease or condition affecting fewer than 200,000 persons in the United States. About 6,000 such disorders have been identified, affecting an estimated 25 million Americans.

The initiative includes interventional trials to test new therapies or drugs, as well as longitudinal or natural history studies that will provide information about the characteristics of rare diseases and their progression over time.
Each consortium in the network includes active participation by the relevant patient advocacy groups. In addition, the Coalition of Patient Advocacy Groups (CPAG) was created to represent the more than 30 patient advocacy groups involved in the network. CPAG has been instrumental in outreach to the affected populations and gaining their input into the development of studies.

The RDCRN is funded by the ORD; NCRR; National Heart, Lung and Blood Institute; National Institute of Child Health and Human Development; National Institute of Neurological Disorders and Stroke; National Institute of Arthritis and Musculoskeletal and Skin Diseases; and National Institute of Diabetes and Digestive and Kidney Diseases-all components of NIH-an agency of the Department of Health and Human Services.

About Seattle Children’s

Consistently ranked as one of the best children’s hospitals in the country by U.S. News & World Report, Seattle Children’s serves as the pediatric and adolescent academic medical referral center for the largest landmass of any children’s hospital in the country (Washington, Alaska, Montana and Idaho). For more than 100 years, Seattle Children’s has been delivering superior patient care while advancing new treatments through pediatric research. Seattle Children’s serves as the primary teaching, clinical and research site for the Department of Pediatrics at the University of Washington School of Medicine. The hospital works in partnership with Seattle Children’s Research Institute and Seattle Children’s Hospital Foundation. For more information, visit www.seattlechildrens.org or follow us on Twitter or Facebook.

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