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

• Asıl Yazarlar: Martine Gehin (Yazar), Meggane Melchior (Yazar), Richard W.D. Welford (Yazar), Patricia N. Sidharta (Yazar), Jasper Dingemanse (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Wiley, 2021-03-01T00:00:00Z.
• Konular: article
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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...