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Columbia University Irving Medical Center with NewYork-Presbyterian has been named a Center of Excellence by the National Organization for Rare Disorders (NORD) and joins a national network dedicated to improving treatment and care for people with rare diseases.

All 31 institutions named as Centers of Excellence have experts across multiple specialties to meet the needs of patients with rare diseases and have a wide range of clinical, patient education, and research programs for people living with rare diseases.

“The history of rare disease at Columbia is profound, with our faculty having discovered the causes of and treatments for many,” says Dr. Jordan Orange, MD, PhD, the Reuben S. Carpentier Professor and Chair of the Department of Pediatrics at Columbia University Vagelos College of Physicians and Surgeons and Physician-in-Chief at NewYork-Presbyterian Morgan Stanley Children’s Hospital. “Those roots live strong, and our present and future are committed to ensuring the same, in concert with the most comprehensive and thoughtful care for patients and their families.”

A disease is considered rare if it affects fewer than 200,000 people. More than 7,000 rare diseases affect 25 million to 30 million American children and adults. More than 90% of rare diseases lack an FDA-approved treatment.

Often, such diseases are not diagnosed until an underlying genetic cause has been identified.

“Columbia and NewYork-Presbyterian have long been recognized as a place where patients and families can go to seek diagnosis and innovative treatment for many rare genetic diseases,” says Dr. Anil Rustgi, MD, Interim Executive Vice President and Dean of the Faculties of Health Sciences and Medicine at Columbia University’s Vagelos College of Physicians and Surgeons/Columbia University Irving Medical Center. “As a NORD Center of Excellence, we will be able to share the collective power of our multidisciplinary clinical expertise, translational research infrastructure, and genetic testing/counseling to help more patients living with rare diseases.”

Dr. Wendy Chung, MD, PhD, the Kennedy Family Professor of Pediatrics (in Medicine) at Columbia University Vagelos College of Physicians and Surgeons, leads the new Center of Excellence at Columbia University Irving Medical Center. As a physician-scientist and clinical geneticist at Columbia University and NewYork-Presbyterian Morgan Stanley Children’s Hospital, she has identified more than 50 rare genetic conditions.

“This is an important time in medicine, when we have the ability to identify more patients with rare diseases, identify what causes the disease, and use precision medicine to find ways to treat them,” Dr. Chung says.

Columbia and NewYork-Presbyterian offer multidisciplinary clinical care for dozens of rare diseases affecting children and adults and have received Center of Excellence designations from other organizations for programs that treat people with spinal muscular atrophy, mitochondrial disorders, and rare cancer syndromes, among others.

“As a NORD Center of Excellence, we’re joining forces with other centers to improve care for all patients with rare diseases, and we will have even more opportunities to participate in clinical trials of new treatments on a much broader scale,” Dr. Chung says.

“Though genetic diseases are individually rare, one in 10 of us has or will have a rare genetic condition, whether we know it or not,” Dr. Chung adds. “There may be as many as 2 million people with rare diseases in the New York City area alone.”

“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,” said Ed Neilan, Chief Scientific and Medical Officer of NORD. “We are proud to announce Columbia University Irving Medical Center 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.”