A: Chondrocyte with constitutionally active FGFR3 that down-regulates its development via the MAPK/ERK pathwayB:<unk>  (BMN 111) blocks this mechanism by binding to the atrial natriuretic peptide receptor B (NPR-B), which subsequently inhibits the MAPK/ERK pathway at the RAF-1 protein.<unk>  works by binding to a receptor (target) called natriuretic peptide receptor type B (NPR-B), which reduces the activity of fibroblast growth factor receptor 3 (FGFR3). FGFR3 is a receptor that normally down-regulates cartilage and bone growth when activated by one of the proteins known as acidic and basic fibroblast growth factor. It does so by inhibiting the development (cell proliferation and differentiation) of chondrocytes, the cells that produce and maintain the cartilaginous matrix which is also necessary for bone growth. Children with achondroplasia have one of several possible FGFR3 mutations resulting in constitutive (permanent) activity of this receptor, resulting in overall reduced chondrocyte activity and thus bone growth. The protein C-type natriuretic peptide (CNP), naturally found in humans, reduces the effects of over-active FGFR3.<unk>  is a CNP analogue with the same effect but prolonged half-life, allowing for once-daily administration.
Vosoritide
Oprelvekin
Selexipag
Eteplirsen
Ganirelix
Tezacaftor
Ecallantide
Vagisil