Article
Temporal order of clinical and biomarker changes in familial frontotemporal dementia.
Nature medicine - 1 Oct 2022
Staffaroni Adam M, Quintana Melanie, Wendelberger Barbara, Heuer Hilary W, Russell Lucy L, Cobigo Yann, Wolf Amy, Goh Sheng-Yang Matt, Petrucelli Leonard, Gendron Tania F, Heller Carolin, Clark Annie L, Taylor Jack Carson, Wise Amy, Ong Elise, Forsberg Leah, Brushaber Danielle, Rojas Julio C, VandeVrede Lawren, Ljubenkov Peter, Kramer Joel, Casaletto Kaitlin B, Appleby Brian, Bordelon Yvette, Botha Hugo, Dickerson Bradford C, Domoto-Reilly Kimiko, Fields Julie A, Foroud Tatiana, Gavrilova Ralitza, Geschwind Daniel, Ghoshal Nupur, Goldman Jill, Graff-Radford Jonathon, Graff-Radford Neill, Grossman Murray, Hall Matthew G H, Hsiung Ging-Yuek, Huey Edward D, Irwin David, Jones David T, Kantarci Kejal, Kaufer Daniel, Knopman David, Kremers Walter, Lago Argentina Lario, Lapid Maria I, Litvan Irene, Lucente Diane, Mackenzie Ian R, Mendez Mario F, Mester Carly, Miller Bruce L, Onyike Chiadi U, Rademakers Rosa, Ramanan Vijay K, Ramos Eliana Marisa, Rao Meghana, Rascovsky Katya, Rankin Katherine P, Roberson Erik D, Savica Rodolfo, Tartaglia M Carmela, Weintraub Sandra, Wong Bonnie, Cash David M, Bouzigues Arabella, Swift Imogen J, Peakman Georgia, Bocchetta Martina, Todd Emily G, Convery Rhian S, Rowe James B, Borroni Barbara, Galimberti Daniela, Tiraboschi Pietro, Masellis Mario, Finger Elizabeth, van Swieten John C, Seelaar Harro, Jiskoot Lize C, Sorbi Sandro, Butler Chris R, Graff Caroline, Gerhard Alexander, Langheinrich Tobias, Laforce Robert, Sanchez-Valle Raquel, de Mendonça Alexandre, Moreno Fermin, Synofzik Matthis, Vandenberghe Rik, Ducharme Simon, Le Ber Isabelle, Levin Johannes, Danek Adrian, Otto Markus, Pasquier Florence, Santana Isabel, Kornak John, Boeve Bradley F, Rosen Howard J, Rohrer Jonathan D, Boxer Adam L
Abstract excerpt
Unlike familial Alzheimer's disease, we have been unable to accurately predict symptom onset in presymptomatic familial frontotemporal dementia (f-FTD) mutation carriers, which is a major hurdle to designing disease prevention trials. We developed multimodal models for f-FTD disease progression and estimated clinical trial sample sizes in C9orf72, GRN and MAPT mutation carriers. Models included longitudinal...