软骨发育不全
医学
身材矮小
成纤维细胞生长因子受体1
内科学
儿科
内分泌学
成纤维细胞生长因子
受体
作者
Ravi Savarirayan,Josep Maria De Bergua,Paul Arundel,Jean‐Pierre Salles,Vrinda Saraff,Borja Delgado,Antonio Leiva‐Gea,Helen McDevitt,Marc Nicolino,Massimiliano Rossi,M. Salcedo,Valérie Cormier‐Daire,Mars Skae,Pekka Kannus,John A. Phillips,Howard M. Saal,Paul Harmatz,Toby Candler,Dawn Hill,Elena Muslimova
标识
DOI:10.1056/nejmoa2411790
摘要
The administration of oral infigratinib did not result in any apparent major safety signal and increased the annualized height velocity and z score and decreased the upper-to-lower body segment ratio at 18 months of treatment in cohort 5. (Funded by BridgeBio Pharma; PROPEL2 ClinicalTrials.gov number, NCT04265651.).
科研通智能强力驱动
Strongly Powered by AbleSci AI