Effect of the Histone Deacetylase Inhibitor FRM-0334 on Progranulin Levels in Patients With Progranulin Gene Haploinsufficiency: A Randomized Clinical Trial
组蛋白去乙酰化酶抑制剂FRM-0334对前粒蛋白基因单倍体不足患者前粒蛋白水平的影响:一项随机临床试验
期刊:Jama Network Open
影响因子:9.7
doi:10.1001/jamanetworkopen.2021.25584
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