Adult Onset Leukoencephalopathy with Axonal Spheroids and Pigmented Glia

Overview

Type of disease: Rare Condition or Disease

ALSP is a rare, inherited, autosomal dominant neurological disease with high penetrance that affects an estimated 10,000 people in the US, with about 1,000 – 2,000 new cases annually. Similar epidemiology exists for Europe and Asia.

The disease generally presents itself in adults in their forties, is diagnosed through genetic testing and established clinical/radiologic criteria and is characterized by cognitive dysfunction, neuropsychiatric symptoms, and motor impairment. These symptoms typically exhibit rapid progression with a life expectancy of approximately six to seven years on average after diagnosis, causing significant patient and caregiver burden. There are currently no approved products for the treatment of ALSP, underlining the high unmet need in this rare indication.

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