U of M Medical School receives $360,000 grant to support development of Krabbe disease registry

MINNEAPOLIS/ST. PAUL (1/24/2025) — The University of Minnesota Medical School recently received a $360,000 grant from the Rosenau Family Research Foundation to establish a registry for Krabbe disease, a rare and life-threatening genetic disorder. This project aims to gather critical data to improve the understanding, diagnosis and treatment of Krabbe disease.

With patient consent, the registry will collect clinical information, genetic data and patient outcomes to support families and researchers. The project is unique because it brings a community-driven approach by collaborating with advocacy groups and families to obtain information, which includes patient progress, challenges and needs. This will help expand knowledge on the variability of the disease process and the anticipated outcomes of the patients affected with Krabbe. 

“In discussions with researchers and clinicians caring for these patients, it became clear that additional information was necessary to better understand this potentially devastating disease. Partnering with the Rosenau Foundation to develop a dedicated Krabbe registry will prove extremely valuable in achieving this goal,” said Paul Orchard, MD, a professor at the U of M Medical School, pediatric blood and bone marrow transplant physician at M Health Fairview and principal investigator of the project. 

By collecting and analyzing data, researchers aim to understand the history of Krabbe disease, enhance the understanding of what to expect through early detection with newborn screenings and ultimately improve decision making and outcomes for patients and families.

Early goals will be to establish the database that will contain the registry data and to develop an electronic mechanism to allow families from across the country to participate in the registry. 

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The University of Minnesota Medical School is at the forefront of learning and discovery, transforming medical care and educating the next generation of physicians. Our graduates and faculty produce high-impact biomedical research and advance the practice of medicine. We acknowledge that the U of M Medical School is located on traditional, ancestral and contemporary lands of the Dakota and the Ojibwe, and scores of other Indigenous people, and we affirm our commitment to tribal communities and their sovereignty as we seek to improve and strengthen our relations with tribal nations. For more information about the U of M Medical School, please visit med.umn.edu.