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A human GBA-L444P transgene drives early and persistent dopamine neurotransmission deficits and alpha-synuclein pathology in a mouse model of early Parkinson’s disease2026 bioRxiv (Cold Spring Harbor Laboratory) preprint Medicine Lysosomal Storage Disorders Research Open access
Natalie Connor‐Robson, Tara Diviney, Javier Alegre‐Abarrategui, Katherine R. Brimblecombe, Nora Bengoa‐Vergniory, Harrison Waters, +7 more
0citations -
A randomized single and multiple ascending dose study in healthy volunteers of LTI‐291, a centrally penetrant glucocerebrosidase activator2021 British Journal of Clinical Pharmacology article Medicine Lysosomal Storage Disorders Research Open access
Jonas M. den Heijer, Annelieke C. Kruithof, Guido van Amerongen, Marieke L. de Kam, Eva Thijssen, Hendrika W. Grievink, +10 more
46citations -
Experience in Genetic Counseling for GBA1 Variants in Parkinson's Disease2020 Movement Disorders Clinical Practice article Medicine Lysosomal Storage Disorders Research Open access
Jonas M. den Heijer, Jacobus Johannes van Hilten, Anneke J.A. Kievit, Vincenzo Bonifati, Geert Jan Groeneveld
10citations
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