Dose selection for biological enzyme replacement therapy indicated for inborn errors of metabolism

• Main Authors: Yuen Yi Hon (Author), Jie Wang (Author), Henrietta Abodakpi (Author), Anand Balakrishnan (Author), Michael Pacanowski (Author), Sushanta Chakder (Author), Patroula Smpokou (Author), Kathleen Donohue (Author), Yow‐Ming C. Wang (Author)
• Format: Book
• Published: Wiley, 2023-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract This paper summarizes key features of the dose‐finding strategies used in the development of 11 approved new molecular entities that are first‐in‐class enzyme replacement therapy (ERT), with a goal to gain insight into the dose exploration approaches to inform efficient dose‐finding in futu...