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

• Principais autores: Martine Gehin (Autor), Meggane Melchior (Autor), Richard W.D. Welford (Autor), Patricia N. Sidharta (Autor), Jasper Dingemanse (Autor)
• Formato: Livro
• Publicado em: Wiley, 2021-03-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: 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...