Since pharmacological targeting of CSF1R with tyrosine kinase inhibitors prevented the disease progression in mouse models of neurodegenerative disorders [2,3,24], a potential pharmacological benefit of CSF1R inhibition remains to be elucidated for patients with HDLS [115]. The gene discussed is CSF1R; the disease is Hereditary diffuse leukoencephalopathy with axonal spheroids and pigmented glia.