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Stem cell transplant trial offers hope for halting rare brain disease

Disease control ENROLLING_BY_INVITATION

This study aims to measure whether stem cell transplants can slow or stop the progression of CSF1R-related leukoencephalopathy, a rare genetic brain disease. Researchers will follow 20 adult patients who have the genetic mutation and are scheduled to receive a transplant. They'll…

Matched conditions: POLD

Sponsor: Mayo Clinic • Aim: Disease control

Last updated Mar 31, 2026 12:11 UTC

POLD

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Stem cell transplant trial offers hope for halting rare brain disease