I am the Director of the Inherited, Metabolic and Storage Disease Transplantation Program at the University of Minnesota. Our group is a world-leading center for the treatment of these disorders by hematopoietic stem cell transplantation, including leukodystrophies, and in particular ALD. My training is in the field of Pediatrics, with subspecialty training in Hematology/Oncology and Blood and Marrow Transplantation. My clinical practice is now focused on the use of hematopoietic stem cell transplantation for non-malignant, inherited disorders, and how we might develop a better understanding the biology of these disorders.
I have extensive experience in the design and implementation of clinical trials for inherited leukodystrophies and I have actively worked to build a team of professionals in various disciplines to provide optimal care for these patients and their families. We are likely the largest center in the world in the treatment of adrenoleukodystrophy. I am also an investigator on the Adrenoleukodystrophy National Registry.
Main Area of Research: Our group maintains the ALD Registry (https://aldnr.umn.edu), and we have a large multidisciplinary clinic to monitor those with ALD, with a focus on boys picked up by newborn screening. We are also one of the largest centers in the world in the treatment of ALD with blood stem cell transplantation, and we have published extensively on these outcomes, biomarkers, neuropsychological issues, etc.