Reference : GBA-associated PD: chances and obstacles for targeted treatment strategies.
Scientific journals : Article
Human health sciences : Neurology
Systems Biomedicine
http://hdl.handle.net/10993/53881
GBA-associated PD: chances and obstacles for targeted treatment strategies.
English
Höglinger, Günter [> >]
Schulte, Claudia [> >]
Jost, Wolfgang H. [> >]
Storch, Alexander [> >]
Woitalla, Dirk [> >]
Krüger, Rejko mailto [University of Luxembourg > Luxembourg Centre for Systems Biomedicine (LCSB) > Translational Neuroscience]
Falkenburger, Björn [> >]
Brockmann, Kathrin [> >]
31-May-2022
Journal of neural transmission (Vienna, Austria : 1996)
129
9
1219-1233
Yes
International
0300-9564
1435-1463
Austria
[en] Cohort Studies ; Glucosylceramidase/genetics ; Humans ; Mutation ; Parkinson Disease/drug therapy/genetics ; GBA ; Lysosomal ; PD ; α-Synuclein
[en] 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.
http://hdl.handle.net/10993/53881
10.1007/s00702-022-02511-7
© 2022. The Author(s).

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