Searchable abstracts of presentations at key conferences on calcified tissues

ba0007oc25 | (1) | ICCBH2019

TransCon CNP: Potential for a once weekly novel therapy in children with achondroplasia

Bharucha Kamal , Ota Sho , Christoffersen Eva Dam , Mygind Per , Viuff Dorthe , Leff Jonathan

Objectives: Achondroplasia (ACH), the most common form of human dwarfism, is caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, a key negative regulator of endochondral ossification. C-type natriuretic peptide (CNP) inhibits the FGFR3 pathway and thereby promotes proliferation and differentiation of chondrocytes to promote bone growth. TransCon CNP is a prodrug designed to provide continuous exposure to CNP to optimize efficacy with ...