Advancing clinical development for neuronopathic Hunter syndrome through a quantitatively‐driven reverse translation framework

• 主要な著者: Robert D. Latzman (著者), Olivia Campagne (著者), Meera E. Modi (著者), Marta Karas (著者), C. J. Malanga (著者), David A. H. Whiteman (著者)
• フォーマット: 図書
• 出版事項: Wiley, 2024-04-01T00:00:00Z.
• 主題: article
• オンライン･アクセス: Connect to this object online.

Abstract A quantitatively‐driven evaluation of existing clinical data and associated knowledge to accelerate drug discovery and development is a highly valuable approach across therapeutic areas, but remains underutilized. This is especially the case for rare diseases for which development is partic...