Natural History of Adult-Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia (ALSP): A Retrospective Patient Cohort Study

This retrospective study provides qualitative and quantitative data from the clinical setting for further characterization of the NH of CSF1R-ALSP, a rare neurological disorder with significant unmet medical need. Data will inform the design of and endpoint selection for future NH studies and interventional clinical trials, which will be central to the development of safe and efficacious therapies for CSF1R-ALSP. Further, it will guide clinicians less familiar with the disease in providing patients and caregivers with appropriate support and information about CSF1R-ALSP.