ey0021.5-11 | Novel Treatments | ESPEYB21
Dauber Andrew
, Zhang Anqing
, Kanakatti Shankar Roopa
, Boucher Kimberly
, McCarthy Tara
, Shafaei Niusha
, Seaforth Raheem
, Grace Castro Meryll
, Dham Niti
, Merchant Nadia
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 ...