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Title: Pharmacotherapy for Frontotemporal Dementia
Authors: Khoury, Rita
Liu, Yu
Sheheryar, Quratulanne
Grossberg, George T
Affiliations: Faculty of Medicine 
Issue Date: 2021
Publisher: Springer
Part of: CNS Drugs
Volume: 35
Issue: 4
Start page: 425
End page: 438
Frontotemporal dementia is a heterogeneous spectrum of neurodegenerative disorders. The neuropathological inclusions are tau proteins, TAR DNA binding protein 43 kDa-TDP-43, or fused in sarcoma-ubiquitinated inclusions. Genetically, several autosomal mutations account for the heritability of the disorder. Phenotypically, frontotemporal dementia can present with a behavioral variant or a language variant called primary progressive aphasia. To date, there are no approved symptomatic or disease-modifying treatments for frontotemporal dementia. Currently used therapies are supported by low-level of evidence (mostly uncontrolled) studies. The off-label use of drugs is also limited by their side-effect profile including an increased risk of confusion, parkinsonian symptoms, and risk of mortality. Emerging disease-modifying treatments currently target the progranulin and the expansion on chromosome 9 open reading frame 72 genes as well as tau deposits. Advancing our understanding of the pathophysiology of the disease and improving the design of future clinical trials are much needed to optimize the chances to obtain positive outcomes.
ISSN: 11727047
DOI: 10.1007/s40263-021-00813-0
Ezproxy URL: Link to full text
Type: Journal Article
Appears in Collections:Faculty of Medicine

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