We were the first in the world to treat a patient with leukodystrophy (globoid cell leukodystrophy, or GLD) with bone marrow transplantation. Since that time, we have treated more than 200 leukodystrophy patients with hematopoietic cell transplant (HCT), commonly known as blood and marrow transplant (BMT), and other therapies. Our team continues to have unrivaled experience treating patients with lysosomal and peroxisomal disorders affecting the nervous system. Learn more about our pioneering research.
We have several clinical trials for ALD patients, and we transplant almost as many patients with cerebral ALD as the rest of the world combined. We have published extensively on the clinical aspects of ALD, including biomarkers, oxidative stress, neuropsychology and imaging. Learn more about our trials related to leukodystrophies.