Kidney and Metabolic Bone Diseases Vol.28 No.3(9)

Theme Pediatric bone : From fetus to adolescent
Title New drugs for pediatric bone diseases
Publish Date 2015/07
Author Keiichi Ozono Department of Developmental Medicine (Pediatrics), Osaka University Graduate School of Medicine
[ Summary ] Several kinds of new treatments, including biological agents, have been developed for skeletal dysplasias and metabolic bone diseases, such as anti-sclerostin antibody for osteogenesis imperfecta and recombinant bone-targeting alkaline phosphatase for hypophosphatasia. Soluble fibroblast growth factor 3 (FGFR3), tyrosine kinase inhibitors, meclozine, C-type natriuretic peptide analogues and statin have been shown to be effective in mice models of chondroplasia via inhibition of FGFR3 signal transduction. A clinical trial of the anti-FGF23 antibody has been conducted for the treatment of patients with FGF23-related hypophosphatemic rickets/osteomalacia. With such developments, we expect drastic improvements in the prognosis of patients with skeletal dysplasias and metabolic bone diseases.
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