GBA-associated PD: chances and obstacles for targeted treatment strategies

Given the clear role of GBA in the pathogenesis of Parkinson’s disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importa...

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Autores principales: Höglinger, Günter, Schulte, Claudia, Jost, Wolfgang H., Storch, Alexander, Woitalla, Dirk, Krüger, Rejko, Falkenburger, Björn, Brockmann, Kathrin
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Vienna 2022
Materias:
Neurology and Preclinical Neurological Studies - Review Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463270/
https://www.ncbi.nlm.nih.gov/pubmed/35639160
http://dx.doi.org/10.1007/s00702-022-02511-7
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author Höglinger, Günter
Schulte, Claudia
Jost, Wolfgang H.
Storch, Alexander
Woitalla, Dirk
Krüger, Rejko
Falkenburger, Björn
Brockmann, Kathrin
author_facet Höglinger, Günter
Schulte, Claudia
Jost, Wolfgang H.
Storch, Alexander
Woitalla, Dirk
Krüger, Rejko
Falkenburger, Björn
Brockmann, Kathrin
author_sort Höglinger, Günter
collection PubMed
description Given the clear role of GBA in the pathogenesis of Parkinson’s disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importantly, differences and characteristics based on mutation severity are recognized, and current as well as potential future treatment options are discussed. These findings will inform future strategies for patient stratification and cohort enrichment as well as suitable outcome measures when designing clinical trials.
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spelling pubmed-94632702022-09-11 GBA-associated PD: chances and obstacles for targeted treatment strategies Höglinger, Günter Schulte, Claudia Jost, Wolfgang H. Storch, Alexander Woitalla, Dirk Krüger, Rejko Falkenburger, Björn Brockmann, Kathrin J Neural Transm (Vienna) Neurology and Preclinical Neurological Studies - Review Article Given the clear role of GBA in the pathogenesis of Parkinson’s disease (PD) and its impact on phenotypical characteristics, this review provides an overview of the current knowledge of GBA-associated PD with a special focus on clinical trajectories and the underlying pathological mechanisms. Importantly, differences and characteristics based on mutation severity are recognized, and current as well as potential future treatment options are discussed. These findings will inform future strategies for patient stratification and cohort enrichment as well as suitable outcome measures when designing clinical trials. Springer Vienna 2022-05-31 2022 /pmc/articles/PMC9463270/ /pubmed/35639160 http://dx.doi.org/10.1007/s00702-022-02511-7 Text en © The Author(s) 2022 https://creativecommons.org/licenses/by/4.0/Open AccessThis article is licensed under a Creative Commons Attribution 4.0 International License, which permits use, sharing, adaptation, distribution and reproduction in any medium or format, as long as you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons licence, and indicate if changes were made. The images or other third party material in this article are included in the article's Creative Commons licence, unless indicated otherwise in a credit line to the material. If material is not included in the article's Creative Commons licence and your intended use is not permitted by statutory regulation or exceeds the permitted use, you will need to obtain permission directly from the copyright holder. To view a copy of this licence, visit http://creativecommons.org/licenses/by/4.0/ (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Neurology and Preclinical Neurological Studies - Review Article
Höglinger, Günter
Schulte, Claudia
Jost, Wolfgang H.
Storch, Alexander
Woitalla, Dirk
Krüger, Rejko
Falkenburger, Björn
Brockmann, Kathrin
GBA-associated PD: chances and obstacles for targeted treatment strategies
title GBA-associated PD: chances and obstacles for targeted treatment strategies
title_full GBA-associated PD: chances and obstacles for targeted treatment strategies
title_fullStr GBA-associated PD: chances and obstacles for targeted treatment strategies
title_full_unstemmed GBA-associated PD: chances and obstacles for targeted treatment strategies
title_short GBA-associated PD: chances and obstacles for targeted treatment strategies
title_sort gba-associated pd: chances and obstacles for targeted treatment strategies
topic Neurology and Preclinical Neurological Studies - Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9463270/
https://www.ncbi.nlm.nih.gov/pubmed/35639160
http://dx.doi.org/10.1007/s00702-022-02511-7
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