Abstract
The skeletal research field continues to develop rapidly and has produced several seminal findings in the last year including advances in the treatment of rare skeletal disorders, and an even deeper understanding into the molecular mechanisms that control skeletal development, endochondral bone formation, mineralization of skeletal tissues and skeletal biology. The targeting of the C-type natriuretic peptide (CNP) pathway and options to directly antagonize the overactivity of the FGFR3 pathway in achondroplasia continues to be a subject of high interest and excitement and we here highlight the double-blind, randomized placebo-controlled phase 3 study of a CNP analogue (vosoritide) in children with achondroplasia. We also highlight the identification of a novel gene for autosomal dominant hypophosphatemic rickets, publication of new growth charts for X-linked hypophosphatemia and two large well-designed paediatric vitamin D trials for the prevention of tuberculosis and asthma exacerbation, respectively.
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