Leukodystrophy Center


We are grateful to our patients, their families, and the advocacy organizations that make our research possible. Together, we continue to make significant progress in the understanding and treatment of leukodystrophies.

Support Our Leukodystrophy Research

Selected Research Projects

One of our goals at the University of Minnesota is to understand the mechanism of how and why the cerebral form of adrenoleukodystrophy (ALD) starts. As the largest transplant center in the world, we are in a unique position to answer this question. Our international experts want to use our biorepository and database to search for potential triggers of cerebral disease. They hope to investigate questions about biomarkers, environmental exposure, gene mutations, imaging, infection, and neuropsychology.

Another goal is to improve the “mouse model” of ALD. Currently, the mice that have the ALD gene mutation do not have cerebral inflammation. We are working on creating a better mouse model of the disease that mimics the cerebral disease of the boys more closely. This will enable us to test new and novel therapies faster and more effectively compared to the current testing performed on cells in a dish.

Paul Orchard and Troy Lund

Run for ALD

The 1st Annual Run for ALD, Cure the Boys was held at Creve Coeur Park in Maryland Heights, MO on November 18, 2017. It was a huge success, with over 300 participants and $10,000 raised for the Adrenoleukodystrophy Research Fund!Run for ALD

Cure the Boys

University of Minnesota groundbreaking gene therapy research

Bridgetown family spreads awareness after boy diagnosed with rare disorder