Cincinnati Children’s Joins NORD Rare Disease Centers of Excellence Network

Post Date: November 4, 2021

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Cincinnati Children’s Hospital Medical Center has been designated as a NORD Rare Disease Center of Excellence, joining 31 medical centers nationwide as part of an innovative network seeking to expand access and advance care and research for people with rare diseases.   

The program, led by the National Organization for Rare Disorders (NORD), seeks to foster knowledge sharing between experts, connect patients to appropriate specialists regardless of disease or geography, and improve the pace of progress in rare disease diagnosis, treatment, and research.

“Clinicians and scientists at Cincinnati Children’s work every day to seek treatments for the many rare diseases that affect our patients,” says John Hogenesch, PhD, Interim Director, Division of Human Genetics. “Especially with the rarest diseases, it’s crucial that research centers work together to share data to improve outcomes. Our hope is that the Centers of Excellence network will accelerate these efforts to catalyze treatments.”

People living with rare diseases frequently face many challenges in finding a diagnosis and quality clinical care. In establishing the Centers of Excellence program, NORD has designated clinical centers across the US that provide exceptional rare disease care and have demonstrated a deep commitment to serving rare disease patients and their families using a holistic, state-of-the-art approach.

Each center was selected by NORD in a competitive application process requiring evidence of staffing with experts across multiple specialties to meet the needs of rare disease patients and significant contributions to rare disease patient education, physician training, and research. The NORD Rare Disease Centers of Excellence program is formulated to achieve better outcomes for all members of the rare disease community. This collaborative partnership strives to push the rare disease field forward by establishing and implementing new standards of care and innovating around new treatments, therapies, and research.

Any disease that affects fewer than 200,000 people in the US is considered rare, according to the National Institutes of Health. There are over 7,000 rare diseases and 25-30 million Americans estimated to be living with rare diseases. More than 90% of rare diseases lack an FDA-approved treatment. 

“Right now, far too many rare diseases are without an established standard of care. The Centers of Excellence program will help set that standard–for patients, clinicians, and medical centers alike,” says Ed Neilan, Chief Scientific and Medical Officer of NORD. “We are proud to announce Cincinnati Children’s as a NORD Rare Disease Center of Excellence and look forward to their many further contributions as we collectively seek to improve health equity, care, and research to support all individuals with rare diseases.”

Cincinnati Children’s has a long history of leadership in rare diseases care and research, including the recent establishment of our Center for Mendelian Genomics and Therapeutics, as well as becoming the first medical center to establish a Center for Circadian Rhythms to treat sleep disorders.

In addition to the new Centers of Excellence designation, the medical center received a five-year $28 million grant in 2019 from the National Center for Advancing Translational Sciences (NCATS) to serve as the data management and coordinating center for the Rare Diseases Clinical Research Network (RDCRN).

For more information on the NORD Rare Disease Centers of Excellence program and the full list of centers, visit the program website.

Learn more about the rare genetic diseases program at Cincinnati Children’s.