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Deficient and Altered Brain White Matter Development in Wolfram Syndrome

Li, Z. A.; Neyman, O.; Rutlin, J.; Lugar, H. M.; Koller, J. M.; Shimony, J. S.; Hershey, T.

2026-05-29 neurology
10.64898/2026.05.27.26354240 medRxiv
Show abstract

Wolfram syndrome (WFS) is characterized by youth-onset insulin-dependent diabetes and neurological deficits. Brain white matter deficiency has been reported, but its trajectory remains unclear. Applying diffusion basis spectrum imaging models longitudinally in 29 individuals with WFS (baseline ages, 5.2 to 25.8 years; maximum 7 visits) and 52 matched controls, we found that WFS is associated with microstructural alterations suggesting diminished axonal integrity, myelin content, and cellularity. These changes were present and stable early in the disease progression in visual and auditory-related regions, whereas abnormalities in the corpus callosum appeared later in adolescence and adulthood. Our results support developmental hypomyelination as a neurophenotype of WFS.

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