This week in therapeutics

Indication

Target/marker/pathway

Summary

Licensing status

Publication and contact ­information

Musculoskeletal disease

Musculoskeletal disease

C-type natriuretic peptide (CNP; NPPC); fibroblast growth factor receptor 3 (FGFR3; CD333)

Ex vivo and mouse studies suggest a CNP analog that antagonizes FGFR3 could help treat achondroplasia, the most common form of dwarfism. In femurs from a mouse model of achondroplasia with a gain-of-function mutation in FGFR3, the CNP analog increased bone length compared with vehicle. In that mouse model, subcutaneous injection of the CNP analog beginning at seven days of age led to increased bone length compared with vehicle injection. In those mice, the CNP injections also corrected skull and growth plate defects. Next steps could include testing the approach in additional models of achondroplasia.

SciBX 5(49); doi:10.1038/scibx.2012.1288
Published online Dec. 20, 2012

Patent and licensing status unavailable

Lorget, F. et al. Am. J. Hum. Genet.; published online Nov. 29, 2012;
doi:10.1016/j.ajhg.2012.10.014
Contact: Laurence Legeai-Mallet, Institut National de la Santé et de la Recherche Médicale (INSERM), Paris, France
e-mail:

laurence.legeai-mallet@inserm.fr