The University of Miami Miller School of Medicine has achieved a prestigious designation, selected as one of the centers in the National Organization for Rare Disorders (NORD) Centers of Excellence program. The program will be hosted under the Division of Clinical and Translational Genetics, where it will be one of the 31 nationwide innovative networks and the only program in Florida that will make up the inaugural cohort.
NORD’s goal with the program, which launches this year, is to have a network of nationwide medical centers seeking to foster knowledge sharing between experts across the country, connect patients to appropriate specialists regardless of disease or geography, and improve the pace of progress in more than 7,000 known rare disorders.
“This is the first example of a center of excellence for all rare conditions or disorders,” said Mustafa Tekin, M.D., chief of the Division of Clinical and Translational Genetics in the John P. Hussman Institute for Human Genomics. “The biggest takeaway from the program will be the vast networking opportunity. While not every physician will be an expert on all 7,000 rare disorders, having a network with such diverse expertise will empower us to seek the best care and find research on a specific condition.”
A Long History
NORD selected each center 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 Division of Clinical and Translational Genetics met and surpassed the criteria, as it already had a long history of leading researchers focusing on finding the cause and understanding the mechanism of these conditions, and having a deep genomic infrastructure with various models. This was further amplified when it became a site for the NIH’s Undiagnosed Diseases Network, which is aimed at accelerating and improving the diagnosis of rare and undiagnosed diseases.
The NORD Rare Disease Centers of Excellence program is formulated to achieve better outcomes for all members of the rare disease community. An estimated 25 to 30 million Americans are living with rare disorders, more than 90% of which lack an FDA-approved treatment.
Setting Standards of Care
“Right now, far too many rare diseases are without an established standard of care,” said Ed Neilan, chief scientific and medical officer of NORD. “The Centers for Excellence program will help set that standard — for patients, clinicians, and medical centers alike. We are proud to announce the Division of Clinical and Translational Genetics 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.”
The Division of Clinical and Translational Genetics will use its affiliation with NORD to build upon its past success and continue to advance treatments for rare genetic conditions, such as geleophysic dysplasia.
“We are in an era in which treatments are being developed,” Dr. Tekin said. “Cures for these disorders were not an option for a long time, and now things are changing and opening up the possibility for a cure to work in this area.”