Frontotemporal dementia

Frontotemporal dementia (FTD) typically presents in the sixth or seventh decade with progressive impairment of executive function, language and social interaction. These symptoms are associated with a characteristic pattern of neurodegeneration affecting the frontal and temporal cortices. In 5-10% of FTD patients, pathogenic loss-of-function mutations can be identified in the gene encoding progranulin, a ubiquitous lysosomal protein. Patients universally exhibit a progressive course, with an average survival of eight years from symptom onset. There are approximately 20,000-30,000 patients with FTD (between the ages of 45-64). There are currently no disease modifying therapies for FTD. The goal of our therapy is to be able to restore CSF PGRN levels to normal. We plan to advance this program into the clinic in 2020.