Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranu...
Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial
About this item
Full title
Author / Creator
Ljubenkov, Peter A , Edwards, Lauren , Iaccarino, Leonardo , La Joie, Renaud , Rojas, Julio C , Koestler, Mary , Harris, Baruch , Boeve, Bradley F , Borroni, Barbara , van Swieten, John C , Grossman, Murray , Pasquier, Florence , Frisoni, Giovanni B , Mummery, Catherine J , Vandenberghe, Rik , Le Ber, Isabelle , Hannequin, Didier , McGinnis, Scott M , Auriacombe, Sophie , Onofrj, Marco , Goodman, Ira J , Riordan, Henry J , Wisniewski, Gary , Hesterman, Jacob , Marek, Ken , Haynes, Beth Ann , Patzke, Holger , Koenig, Gerhard , Hilt, Dana , Moebius, Hans and Boxer, Adam L
Publisher
United States: American Medical Association
Journal title
Language
English
Formats
Publication information
Publisher
United States: American Medical Association
Subjects
More information
Scope and Contents
Contents
Histone deacetylase inhibitors have been repeatedly shown to elevate progranulin levels in preclinical models. This report describes the first randomized clinical trial of a histone deacetylase inhibitor in frontotemporal dementia (FTD) resulting from progranulin (GRN) gene variations.
To characterize the safety, tolerability, plasma pharmacokin...
Alternative Titles
Full title
Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial
Authors, Artists and Contributors
Author / Creator
Edwards, Lauren
Iaccarino, Leonardo
La Joie, Renaud
Rojas, Julio C
Koestler, Mary
Harris, Baruch
Boeve, Bradley F
Borroni, Barbara
van Swieten, John C
Grossman, Murray
Pasquier, Florence
Frisoni, Giovanni B
Mummery, Catherine J
Vandenberghe, Rik
Le Ber, Isabelle
Hannequin, Didier
McGinnis, Scott M
Auriacombe, Sophie
Onofrj, Marco
Goodman, Ira J
Riordan, Henry J
Wisniewski, Gary
Hesterman, Jacob
Marek, Ken
Haynes, Beth Ann
Patzke, Holger
Koenig, Gerhard
Hilt, Dana
Moebius, Hans
Boxer, Adam L
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8463943
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_8463943
Other Identifiers
ISSN
2574-3805
E-ISSN
2574-3805
DOI
10.1001/jamanetworkopen.2021.25584
