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

• मुख्य लेखकों: Martine Gehin (लेखक), Meggane Melchior (लेखक), Richard W.D. Welford (लेखक), Patricia N. Sidharta (लेखक), Jasper Dingemanse (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Wiley, 2021-03-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: Connect to this object online.

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...