Real-World Evidence on the Effectiveness and Safety of Vosoritide in Latin American Patients With Achondroplasia (EVOLAC).
Achondroplasia is a skeletal dysplasia associated with severe short stature and multisystem complications due to a gain-of-function pathogenic variant in the FGFR3 gene. Vosoritide, a C-type natriuretic peptide analog, has demonstrated efficacy in clinical trials. The objective of this study was to describe the effecti...