Assessment of Target Engagement in a First‐in‐Human Trial with Sinbaglustat, an Iminosugar to Treat Lysosomal Storage Disorders

In this first‐in‐human study, the tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple oral doses of sinbaglustat, a dual inhibitor of glucosylceramide synthase (GCS) and non‐lysosomal glucosyl ceramidase (GBA2), were investigated in healthy subjects. The single‐asce...

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Detalles Bibliográficos
Autores principales: Gehin, Martine, Melchior, Meggane, Welford, Richard W.D., Sidharta, Patricia N., Dingemanse, Jasper
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2020
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7993281/
https://www.ncbi.nlm.nih.gov/pubmed/33142037
http://dx.doi.org/10.1111/cts.12911

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