A randomized single and multiple ascending dose study in healthy volunteers of LTI‐291, a centrally penetrant glucocerebrosidase activator

AIMS: A mutation in the GBA1 gene is the most common genetic risk factor for developing Parkinson's disease. GBA1 encodes the lysosomal enzyme glucosylceramidase beta (glucocerebrosidase, GCase) and mutations decrease enzyme activity. LTI‐291 is an allosteric modulator of GCase, enhancing its a...

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Detalles Bibliográficos
Autores principales: den Heijer, Jonas M., Kruithof, Annelieke C., van Amerongen, Guido, de Kam, Marieke L., Thijssen, Eva, Grievink, Hendrika W., Moerland, Matthijs, Walker, Mike, Been, Kees, Skerlj, Renato, Justman, Craig, Dudgeon, Lindsay, Lansbury, Peter, Cullen, Valerie C., Hilt, Dana C., Groeneveld, Geert Jan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8451761/
https://www.ncbi.nlm.nih.gov/pubmed/33576113
http://dx.doi.org/10.1111/bcp.14772

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