@article{85e43debd4754f2b956a136111614168,
title = "GBA-associated PD: chances and obstacles for targeted treatment strategies",
abstract = "Given the clear role of GBA in the pathogenesis of Parkinson{\textquoteright}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.",
keywords = "GBA, Lysosomal, PD, α-Synuclein",
author = "G{\"u}nter H{\"o}glinger and Claudia Schulte and Jost, {Wolfgang H.} and Alexander Storch and Dirk Woitalla and Rejko Kr{\"u}ger and Bj{\"o}rn Falkenburger and Kathrin Brockmann",
note = "Funding Information: Kathrin Brockmann has received a research grants from the University of Tuebingen (Clinician Scientist), the German Society of Parkinson{\textquoteright}s disease (dpv), the Michael J. Fox Foundation (MJFF), the German Centre for Neurodegenerative Diseases (DZNE, MIGAP), and the German Federal Ministry of Education and Research (BMBF) in the frame of ERACoSysMed2 (FKZ 031L0137B). She received Speaker honoraria from Abbvie, Lundbeck, UCB, and Zambon. Funding Information: G{\"u}nther H{\"o}glinger was supported by Deutsche Forschungsgemeinschaft (DFG, German Research Foundation) under Germany{\textquoteright}s Excellence Strategy within the framework of the Munich Cluster for Systems Neurology (EXC 2145 SyNergy—ID 390857198) and within the Hannover Cluster RESIST (EXC 2155—ID 390874280), DFG grants (HO2402/6-2, HO2402/18-1 MSAomics), the German Federal Ministry of Education and Research (BMBF, 01KU1403A EpiPD; 01EK1605A HitTau; 01DH18025 TauTherapy), the German Center for Neurodegenerative Diseases e.V. (DZNE), Nieders{\"a}chsisches Ministerium f{\"u}r Wissenschaft und Kunst (MWK, ZN3440.TP): REBIRTH—Forschungszentrum f{\"u}r translationale regenerative Medizin; VolkswagenStiftung (Nieders{\"a}chsisches Vorab); Petermax-M{\"u}ller Foundation (Etiology and Therapy of Synucleinopathies and Tauopathies); the German Parkinson Society (DPG; ProAPS), the German PSP Association (PSP Gesellschaft; ProPSP); participated in industry-sponsored research projects from Abbvie, Biogen, Biohaven, Novartis, Roche, Sanofi, UCB. Claudia Schulte has nothing to disclose. Wolfgang H. Jost is/was speaker and/or advisor Abbvie, Bial, Kyowa Kirin, Merz, UCB, Zambon. Alexander Storch has received funding from the Deutsche Forschungsgemeinschaft (DFG) and the Helmholtz-Association. He received honoraria for presentations/lectures/consultancies or advisory boards from AbbVie, Bayer Healthcare, Desitin, Bial, GKC, Gr{\"u}nenthal, UCB, Zambon, Ca, AbbVie, TEVA, Lundbeck, and UCB Pharma, outside the submitted work. He has served on the editorial boards of Stem Cells and Stem Cells International and received royalties from Kohlhammer Verlag and Elsevier Press. Dirk Woitalla has nothing to disclose. Rejko Kr{\"u}ger has nothing to disclose. Bj{\"o}rn Falkenburger has nothing to disclose. Publisher Copyright: {\textcopyright} 2022, The Author(s).",
year = "2022",
month = sep,
doi = "10.1007/s00702-022-02511-7",
language = "English",
volume = "129",
pages = "1219--1233",
journal = "Journal of Neural Transmission",
issn = "0300-9564",
publisher = "Springer",
number = "9",
}