Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery
本研究旨在探讨沃索利肽在有颈髓减压手术风险的软骨发育不全婴幼儿中的安全性，并阐述一项随机、对照、开放标签临床试验（含开放标签扩展试验）的理论依据、设计和方法。
期刊:Science Progress
影响因子:2.9
doi:10.1177/00368504211003782
Savarirayan, Ravi; Irving, Melita; Maixner, Wirginia; Thompson, Dominic; Offiah, Amaka C; Connolly, Daniel Ja; Raghavan, Ashok; Powell, James; Kronhardt, Marcin; Jeha, George; Ghani, Sajda; Fisheleva, Elena; Day, Jonathan Rs
