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M Health Fairview Masonic Children’s Hospital recognized as Rare Disease Center of Excellence
Our children’s hospital joins a group of 31 centers nationwide recognized by the National Organization for Rare Disorders (NORD) for unparalleled commitment to rare disease care.
- November 04, 2021
- By Staff Writer
M Health Fairview Masonic Children’s Hospital has been named a Rare Disease Center of Excellence by the National Organization for Rare Disorders (NORD). We join a group of only 31 centers nationwide committed to expanding access and advancing care and research for patients with rare diseases.
In the United States, a disease is defined as “rare” if it affects fewer than 200,000 people. There are more than 7,000 known rare diseases While each unique condition may be rare, together they affect millions of people and their families. In the United States alone, 25 to 30 million people are thought to be living with a rare disease.
Patients with a rare disease often struggle to receive an accurate diagnosis and quality clinical care. NORD has designated Centers of Excellence nationwide that have shown a deep commitment to serving rare disease patients using a state-of-the-art approach. Each center is selected through a competitive application process, based on evidence of expertise across multiple specialties, contributions to patient education, physician training, and research.
While a variety of factors may cause or contribute to rare diseases, many conditions are believed to be genetic. This means they are caused by changes in genes or chromosomes, sometimes passed from one generation to the next. Treatment for many genetic conditions involves blood and marrow transplantation. Our health system performed the first human bone marrow transplant from a matched donor in 1968. Since then, patients from around the world have come to us for leading-edge care.
“We’re proud to serve the thousands of Minnesota children living with a rare disease, as well as patients from around the globe,” said Pediatric Hematologist/Oncologist Joseph Neglia, MD, MPH, Physician-In-Chief at M Health Fairview Masonic Children’s Hospital and Head of the Department of Pediatrics at University of Minnesota Medical School. “We aim to provide holistic care for patients and their families, working with them to advance the frontier of rare disease treatment.”
After leading the way in blood and marrow transplantation, many now turn to M Health Fairview to lead the way in gene therapy. This new treatment has the potential to correct underlying genetic defects, which can lead to a cure for many rare diseases, rather than simply managing symptoms.
“Not only are we advancing life-changing treatments through research, we’re making these new therapies available to patients as Minnesota’s only academic children’s hospital,” said M Health Fairview Geneticist Susan Berry, MD, FAAP, FACMG. Berry is also a professor in the Department of Pediatrics at University of Minnesota Medical School.