Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial

IMPORTANCE: 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. O...

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Detalles Bibliográficos
Autores principales: 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, Boxer, Adam L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2021
Materias:
Original Investigation
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8463943/
https://www.ncbi.nlm.nih.gov/pubmed/34559230
http://dx.doi.org/10.1001/jamanetworkopen.2021.25584

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