C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia
Autoři | |
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Rok publikování | 2019 |
Druh | Článek v odborném periodiku |
Časopis / Zdroj | New England Journal of Medicine |
Fakulta / Pracoviště MU | |
Citace | |
www | http://dx.doi.org/10.1056/NEJMc1910394 |
Doi | http://dx.doi.org/10.1056/NEJMc1910394 |
Klíčová slova | Achondroplasia; C-type natriuretic peptide |
Popis | 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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