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This database contains 1 studies, archived under the term: "progranulin"

Results from a pilot study on amiodarone administration in monogenic frontotemporal dementia with granulin mutation
Frontotemporal dementia (FTD) is one of the most important neurodegenerative conditions and Granulin (GRN) is one of the major genes associated to the disease. FTD-GRN patients are still orphan for any evidence-based target-therapy approach. Interestingly, it has been recently found that alkalizing agents rescued haploinsufficiency in cellular models expressing FTD-GRN mutations. We set up a […]

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