ESPE Yearbook of Paediatric Endocrinology

In brief: This article reviews the evidence and provides expert opinion on the safe and appropriate use of denosumab in children and adolescents with RANKL-mediated disorders such as giant cell bone tumours, fibrous dysplasia and juvenile Paget’s disease.Commentary: Receptor activator of nuclear factor κB ligand (RANKL) is expressed by osteogenic cells and induces osteoclast differentiation by binding to RANK on osteoclast precursors. Excessive...

In brief: This single-arm, open-label, single-centre, Phase 2 study in the US examined the safety and efficacy of vosoritide, a recombinant C-type natriuretic peptide (CNP) analogue, in 26 children with hypochondroplasia. Height SDS increased by (mean) 0.36 during the 12-month treatment period versus the observation period. The study was sponsored by BioMarin Pharmaceutical.Commentary: Hypochondroplasia is a rare skeletal dysplasia that manifests mainly ...