C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia

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Authors

KREJČÍ Pavel

Year of publication 2019
Type Article in Periodical
Magazine / Source New England Journal of Medicine
MU Faculty or unit

Faculty of Medicine

Citation
Web http://dx.doi.org/10.1056/NEJMc1910394
Doi http://dx.doi.org/10.1056/NEJMc1910394
Keywords Achondroplasia; C-type natriuretic peptide
Description Savarirayan et al. (July 4 issue)1 report increased skeletal growth in patients with achondroplasia (a common type of dwarfism caused by activating mutations in the tyrosine kinase fibroblast growth factor receptor 3 gene FGFR3) who received vosoritide, a stable analogue of C-type natriuretic peptide. Their study is a breakthrough in treatment for achondroplasia, and it offers an intriguing possibility of broader clinical applications.
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