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@ARTICLE{Hermann:281785,
author = {Hermann, Andreas and Prudlo, Johannes and Kasper, Elisabeth
and Synofzik, Matthis and Peters, Oliver and Priller, Josef
and Dinter, Elisabeth and Wiltfang, Jens and Zerr, Inga and
Flöel, Agnes and Bürger, Katharina and Höglinger, Günter
U and Levin, Johannes and Düzel, Emrah and Teipel, Stefan
and Beichert, Lukas and Brosseron, Frederic and Wagner,
Michael and Frommann, Ingo and Ramirez, Alfredo and Yakupov,
Renat and Schmid, Matthias and Lingor, Paul and Haass,
Christian and DESCRIBE-ALS and Spottke, Annika and Günther,
René and Weydt, Patrick and Neumann, Manuela and Schneider,
Anja},
collaboration = {consortium, DESCRIBE-FTD},
title = {'{T}he {DESCRIBE}-{ALS}-{FTD} study: a prospective
multicenter observational study of the {ALS}-{FTD}
spectrum'.},
journal = {Amyotrophic lateral sclerosis $\&$ frontotemporal
degeneration},
volume = {26},
number = {7-8},
issn = {2167-8421},
address = {Abingdon},
publisher = {Taylor Francis Group},
reportid = {DZNE-2025-01178},
pages = {720 - 728},
year = {2025},
abstract = {Background: Amyotrophic lateral sclerosis (ALS) and
frontotemporal dementia (FTD) exhibit significant clinical,
genetic and neuropathological abnormalities, and are
regarded as belonging to a common disease spectrum, referred
to as the ALS-FTD spectrum disorders. Our understanding of
the underlying mechanisms of these diseases has advanced
significantly, including molecular neuropathology, genetics
and molecular pathophysiology. The heterogeneity of these
diseases poses significant challenges to translational
research and drug development, particularly in sporadic
cases. Consequently, there is an urgent need to improve
patient stratification for the successful execution of
future clinical trials. Methods/Results: We here describe
the study design of the DESCRIBE-ALS/FTD study which aims to
address this research gap by undertaking a systematic
sampling of patients from the ALS FTD spectrum, encompassing
all possible disease variants. The main objective of the
study is to systematically document detailed cross-sectional
phenotyping and the temporal progression of motor and
neuropsychological abnormalities that occur in both ALS and
FTD. Additionally, it seeks to systematically correlate
these abnormalities with genetics and potentially predictive
biomarkers including longitudinal biomaterial sampling,
brain imaging and brain banking. Furthermore, first-degree
relatives of patients with disease-causing gene variants
undergo the same assessments to also sample presymptomatic
risk gene carriers. Conclusion: With this prospective
registry study we aim to generate datasets which will help
researchers identifying different disease traits in people
with sporadic and genetic ALS and FTD and to develop
biomarkers to identify preclinical and prodromal disease
stages.},
keywords = {Humans / Amyotrophic Lateral Sclerosis: genetics /
Amyotrophic Lateral Sclerosis: diagnosis / Amyotrophic
Lateral Sclerosis: physiopathology / Frontotemporal
Dementia: genetics / Frontotemporal Dementia: diagnosis /
Prospective Studies / Male / Female / Middle Aged / Aged /
Disease Progression / Cross-Sectional Studies / Amyotrophic
lateral sclerosis (Other) / biomarkers (Other) / disease
traits (Other) / frontotemporal dementia (Other) / genetics
(Other) / neuropathology (Other)},
cin = {AG Schneider / AG Hermann / AG Teipel / AG Gasser / AG
Peters / AG Priller / AG Falkenburger / AG Wiltfang / AG
Zerr / AG Flöel / Clinical Research (Munich) / AG Levin /
AG Düzel / AG Heneka / AG Wagner / Patient Studies (Bonn) /
AG Schmid Bonn / AG Haass / AG Spottke / Clinical Research
Platform (CRP) / Clinical Research (Bonn) / AG Neumann},
ddc = {610},
cid = {I:(DE-2719)1011305 / I:(DE-2719)1511100 /
I:(DE-2719)1510100 / I:(DE-2719)1210000 / I:(DE-2719)5000000
/ I:(DE-2719)5000007 / I:(DE-2719)1710012 /
I:(DE-2719)1410006 / I:(DE-2719)1440011-1 /
I:(DE-2719)5000081 / I:(DE-2719)1111015 / I:(DE-2719)1111016
/ I:(DE-2719)5000006 / I:(DE-2719)1011303 /
I:(DE-2719)1011201 / I:(DE-2719)1011101 / I:(DE-2719)1013028
/ I:(DE-2719)1110007 / I:(DE-2719)1011103 /
I:(DE-2719)1011401 / I:(DE-2719)1011001 /
I:(DE-2719)1210003},
pnm = {353 - Clinical and Health Care Research (POF4-353) / 352 -
Disease Mechanisms (POF4-352)},
pid = {G:(DE-HGF)POF4-353 / G:(DE-HGF)POF4-352},
experiment = {EXP:(DE-2719)DESCRIBE-FTD-20160101},
typ = {PUB:(DE-HGF)16},
pubmed = {pmid:40488385},
doi = {10.1080/21678421.2025.2509617},
url = {https://pub.dzne.de/record/281785},
}