Article (Scientific journals)
GBA-associated PD: chances and obstacles for targeted treatment strategies.
Höglinger, Günter; Schulte, Claudia; Jost, Wolfgang H. et al.
2022In Journal of neural transmission (Vienna, Austria : 1996), 129 (9), p. 1219-1233
Peer reviewed
 

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Keywords :
Cohort Studies; Glucosylceramidase/genetics; Humans; Mutation; Parkinson Disease/drug therapy/genetics; GBA; Lysosomal; PD; α-Synuclein
Abstract :
[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.
Disciplines :
Neurology
Author, co-author :
Höglinger, Günter
Schulte, Claudia
Jost, Wolfgang H.
Storch, Alexander
Woitalla, Dirk
Krüger, Rejko ;  University of Luxembourg > Luxembourg Centre for Systems Biomedicine (LCSB) > Translational Neuroscience
Falkenburger, Björn
Brockmann, Kathrin
External co-authors :
yes
Language :
English
Title :
GBA-associated PD: chances and obstacles for targeted treatment strategies.
Publication date :
31 May 2022
Journal title :
Journal of neural transmission (Vienna, Austria : 1996)
ISSN :
0300-9564
eISSN :
1435-1463
Volume :
129
Issue :
9
Pages :
1219-1233
Peer reviewed :
Peer reviewed
Focus Area :
Systems Biomedicine
Commentary :
© 2022. The Author(s).
Available on ORBilu :
since 16 January 2023

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