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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Bibliographic Details
Main Authors: Martine Gehin (Author), Meggane Melchior (Author), Richard W.D. Welford (Author), Patricia N. Sidharta (Author), Jasper Dingemanse (Author)
Format: Book
Published: Wiley, 2021-03-01T00:00:00Z.
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3rd Floor Main Library

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Call Number: A1234.567
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