中国临床药理学杂志2025，Vol.41Issue(22)：3284-3289,6.DOI:10.13699/j.cnki.1001-6821.2025.22.020
营养不良型大疱性表皮松解症先进治疗药品临床试验设计考量
Clinical trial design considerations for advanced therapy medicinal products in dystrophic epidermolysis bullosa
摘要
Abstract
Dystrophic epidermolysis bullosa(DEB)is a mechanobullous disorder caused by mutations in the COL7A1 gene encoding type Ⅶ collagen.It has been included in China's Rare Disease List,with clinical management primarily focused on preventing mechanical trauma and infections.In recent years,significant progress has been made in the development of advanced therapy medicinal products(ATMPs)for DEB,with two products already approved for marketing and multiple others undergoing clinical trials.This article reviews the fundamental characteristics of both approved and investigational ATMPs for DEB and discusses key considerations in clinical trial design,aiming to provide valuable insights for the clinical development of advanced therapies for this condition.关键词
先进治疗药品/营养不良型大疱性表皮松解症/基因治疗/细胞治疗/临床试验设计Key words
advanced therapeutic medicinal products/dystrophic epidermolysis bullosa/gene therapy/cell therapy/clinical trial design分类
医药卫生引用本文复制引用
赵晨阳,鲁爽..营养不良型大疱性表皮松解症先进治疗药品临床试验设计考量[J].中国临床药理学杂志,2025,41(22):3284-3289,6.基金项目
药品监管科学体系建设重点基金项目(RS2024S003) （RS2024S003）
