COLUMBUS, Ohio - Nationwide Children’s Hospital has been selected as a Rare Disease Center of Excellence by the National Organization for Rare Disorders (NORD), a nonprofit patient advocacy organization committed to the identification, treatment and cure of rare disorders through education, research and patient services. This designation recognizes Nationwide Children’s as a trusted medical “home,” where patients with rare diseases and their families can find answers and highlights the expertise of our clinicians and researchers. The hospital joins a highly select group of 31 medical centers, nationwide, as part of an innovative network seeking to expand access and advance care and research for rare disease patients in the United States.
This designation also recognizes work of Kim L McBride, MD, MS, and his clinical group, the Division of Genetic and Genomic Medicine, in particular co-applicant Dr. Mari Mori, to investigate and treat the genetic and genomic causes of rare disorders. In collaboration with the Steve and Cindy Rasmussen Institute for Genomic Medicine, Dr. McBride’s lab has helped expand the phenotype of known disease genes and identify new disease genes for congenital heart disease, inborn errors of metabolism, lethal skeletal dysplasias, movement disorders, osteopenia, cardiomyopathy and other rare diseases. Once genomic testing identifies the cause, the Division also leads the way to rapidly bring novel treatments to those with rare disorders, through our new Therapeutics and Lysosomal Storage Disease Clinic and involvement in clinical trials.
“We are thrilled to be recognized by NORD as a Center of Clinical Excellence for the care of those with rare disorders,” said Dr. McBride, Division Chief of Genetic and Genomic Medicine at Nationwide Children's Hospital. “Nationwide Children’s Hospital has made genomics, with an emphasis on rare disorders, a key part of the five-year Strategic Plan. This designation highlights our abilities not only to diagnose, but to improve the care of those with rare disorders.”
As part of the Rare Disease Centers of Excellence network, Nationwide Children’s will engage in collaborative efforts to improve standards of care, advance state-of-the-art rare disease research and treatment and increase awareness about rare diseases in the broader medical and patient communities.
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 currently 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 for Excellence program will help set that standard – for patients, clinicians, and medical centers alike,” said Ed Neilan, Chief Scientific and Medical Officer of NORD. “We are proud to announce Nationwide 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.”
For more information on the NORD Rare Disease Centers of Excellence program and the full list of centers, visit the program website.