ey0021.5-12 | Novel Treatments | ESPEYB21
Savarirayan Ravi
, Hoernschemeyer Daniel G.
, Ljungberg Merete
, Zarate Yuri A.
, Bacino Carlos A.
, Bober Michael B.
, Legare Janet M.
, et al.
In brief: This multinational, randomised, double-blind, placebo-controlled, dose-escalation Phase 2 study evaluated the safety and efficacy of TransCon CNP (navepegritide), a C-type natriuretic peptide (CNP) analogue designed to allow continuous CNP exposure with once-weekly dosing, in 42 children with achondroplasia aged 2-10 years. The study was sponsored by Ascendis Pharma.Commentary: Achondroplasia is one of the most common constitutional bone disord...