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

Research output: Contribution to journalReview articleInvitedpeer-review


  • Günter Höglinger - , Hannover Medical School (MHH) (Author)
  • Claudia Schulte - , University Hospital Tübingen (Author)
  • Wolfgang H Jost - , Parkinson-Klinik Ortenau (Author)
  • Alexander Storch - , University of Rostock (Author)
  • Dirk Woitalla - , St. Josef Hospital GmbH Moers (Author)
  • Rejko Krüger - , Luxembourg Institute of Health (Author)
  • Björn Falkenburger - , Department of Neurology (Author)
  • Kathrin Brockmann - , University Hospital Tübingen (Author)


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.


Original languageEnglish
Pages (from-to)1219-1233
Number of pages15
JournalJournal of neural transmission
Issue number9
Publication statusPublished - Sept 2022

External IDs

PubMedCentral PMC9463270
Scopus 85131102002
ORCID /0000-0002-2387-526X/work/150328949



  • Cohort Studies, Glucosylceramidase/genetics, Humans, Mutation, Parkinson Disease/drug therapy