This page lists available research studies for individuals with known genetic variants in the progranulin gene (GRN). Please click on the links below to learn more about active clinical trials.
The site is provided for informational purposes only, and is not intended to provide, or be a substitute for, medical advice, diagnosis or treatment recommendations. See full disclaimer.
The Site is provided for informational purposes only, and is not intended to provide, or be a substitute for, medical advice, diagnosis or treatment recommendations. See full disclaimer.
Summary: VES001 is an experimental treatment for healthy individuals with a known pathogenic progranulin gene (GRN) variant designed to restore normal levels of the progranulin protein (PGRN) in the brain.
Summary: AVB-101 is an experimental gene therapy for with people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: DNL593 is an experimental treatment that was designed to restore normal levels of the progranulin protein (PGRN) in the brain in people with symptoms related to FTD who also have a genetic variant in the progranulin gene (GRN).
Summary: PBFT02 is an experimental gene therapy for people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: LY3884963 is being developed as a single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).
Summary: AVB-101 is an experimental gene therapy for with people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: PBFT02 is an experimental gene therapy for people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: LY3884963 is being developed as a single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).
Summary: AVB-101 is an experimental gene therapy for with people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: DNL593 is an experimental treatment that was designed to restore normal levels of the progranulin protein (PGRN) in the brain in people with symptoms related to FTD who also have a genetic variant in the progranulin gene (GRN).
Summary: PBFT02 is an experimental gene therapy for people with frontotemporal dementia and genetic variants in the progranulin gene (FTD-GRN).
Summary: LY3884963 is being developed as a single-dose gene therapy for patients with frontotemporal dementia with GRN mutations (FTD-GRN).
Summary: VES001 is an experimental treatment for healthy individuals with a known pathogenic progranulin gene (GRN) variant designed to restore normal levels of the progranulin protein (PGRN) in the brain.
Summary: AL001 is an experimental treatment designed to slow the progression of Frontotemporal Dementia in individuals with a known pathogenic progranulin gene (GRN) variant. This study is closed to enrollment after meeting its target enrollment.
Note: This page is specifically focused on clinical trials for individuals with FTD caused by progranulin genetic variants. To find out if you are eligible for free genetic counseling and genetic testing click here.
To find clinical trials related to other forms of FTD please visit: www.clinicaltrials.gov
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