Ravi Savarirayan, William R Wilcox, Paul Harmatz, John Phillips, Lynda E Polgreen, Louise Tofts, Keiichi Ozono, Paul Arundel, Melita Irving, Carlos A Bacino, Donald Basel, Michael B Bober, Joel Charrow, Hiroshi Mochizuki, Yumiko Kotani, Howard M Saal, Clare Army, George Jeha, Yulan Qi, Lynn Han, Elena Fisheleva, Alice Huntsman-Labed, Jonathan Day
BACKGROUND: Vosoritide is a recombinant C-type natriuretic peptide analogue that increases annualised growth velocity in children with achondroplasia aged 5-18 years. We aimed to assess the safety and efficacy of vosoritide in infants and children younger than 5 years. METHODS: This double-blind, randomised, placebo-controlled, phase 2 trial was done in 16 hospitals across Australia, Japan, the UK, and the USA. Children younger than 60 months with a clinical diagnosis of achondroplasia confirmed by genetic testing and who had completed a baseline growth study or observation period were enrolled into one of three sequential cohorts based on age at screening: 24-59 months (cohort 1); 6-23 months (cohort 2); and 0-5 months (cohort 3)...
November 17, 2023: Lancet Child & Adolescent Health