Leukoencephalopathy, diffuse hereditary, with spheroids 1
MONDO:0800027A rare autosomal dominant disease characterized by a complex phenotype including progressive dementia, apraxia, apathy, impaired balance, parkinsonism, spasticity and epilepsy.
Also known as: HDLS, hereditary diffuse leukoencephalopathy with spheroids, leukoencephalopathy, diffuse hereditary, with spheroids, leukoencephalopathy, hereditary diffuse, with spheroids, ALSP, Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia, CSF1R-related ALSP, CSF1R-related adult-onset leukoencephalopathy with axonal spheroids and pigmented glia
6 clinical trials for this condition and its sub-types, 6 tagged with Leukoencephalopathy, diffuse hereditary, with spheroids 1 itself.
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Stem cell hope for rare brain disease: 20 patients tracked
Disease control By invitation onlyThis study follows 20 adults with CSF1R-related leukoencephalopathy, a rare genetic brain disease, who are scheduled for a stem cell transplant. Researchers will measure changes in thinking, movement, and brain scans over time to see if the transplant helps stabilize or improve s…
Sponsor: Mayo Clinic • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC
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New stem cell approach aims to tame rare genetic diseases
Disease control OngoingThis study tests a stem cell transplant method for people with inherited metabolic disorders and severe osteopetrosis. The goal is to get the donor cells to take hold while keeping side effects low. Participants receive chemotherapy drugs before the transplant to prepare their bo…
Phase 2 • Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jun 27, 2026 08:09 UTC