An 8-week, open-label, dose-finding study of nimodipine for the treatment of progranulin insufficiency from GRN gene mutations.
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ABSTRACT:
SUBMITTER: Sha SJ
PROVIDER: S-EPMC5671622 | biostudies-literature | 2017 Nov
REPOSITORIES: biostudies-literature
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An 8-week, open-label, dose-finding study of nimodipine for the treatment of progranulin insufficiency from <i>GRN</i> gene mutations.
Sha Sharon J SJ Miller Zachary A ZA Min Sang-Won SW Zhou Yungui Y Brown Jesse J Mitic Laura L LL Karydas Anna A Koestler Mary M Tsai Richard R Corbetta-Rastelli Chiara C Lin Sophie S Hare Emma E Fields Scott S Fleischmann Kirsten E KE Powers Ryan R Fitch Ryan R Martens Lauren Herl LH Shamloo Mehrdad M Fagan Anne M AM Farese Robert V RV Pearlman Rodney R Seeley William W Miller Bruce L BL Gan Li L Boxer Adam L AL
Alzheimer's & dementia (New York, N. Y.) 20170912 4
<h4>Introduction</h4>Frontotemporal lobar degeneration-causing mutations in the progranulin (<i>GRN</i>) gene reduce progranulin protein (PGRN) levels, suggesting that restoring PGRN in mutation carriers may be therapeutic. Nimodipine, a Food and Drug Administration-approved blood-brain barrier-penetrant calcium channel blocker, increased PGRN levels in PGRN-deficient murine models. We sought to assess safety and tolerability of oral nimodipine in human <i>GRN</i> mutation carriers.<h4>Methods</ ...[more]
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