دورية أكاديمية
Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
| العنوان: | Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study |
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| المؤلفون: | Savarirayan, R, Tofts, L, Irving, M, Wilcox, WR, Bacino, CA, Hoover-Fong, J, Font, RU, Harmatz, P, Rutsch, F, Bober, MB, Polgreen, LE, Ginebreda, I, Mohnike, K, Charrow, J, Hoernschemeyer, D, Ozono, K, Alanay, Y, Arundel, P, Kotani, Y, Yasui, N, White, KK, Saal, HM, Leiva-Gea, A, Luna-Gonzalez, F, Mochizuki, H, Basel, D, Porco, DM, Jayaram, K, Fisheleva, E, Huntsman-Labed, A, Day, JRS |
| بيانات النشر: | SPRINGERNATURE Nature Publishing Group US |
| سنة النشر: | 2021 |
| مصطلحات موضوعية: | envir, socio |
| الوصف: | PURPOSE: Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported. METHODS: After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day. RESULTS: In children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104. In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104. No new adverse effects of vosoritide were detected. CONCLUSION: Vosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | http://hdl.handle.net/11343/287664Test |
| الإتاحة: | http://hdl.handle.net/11343/287664Test |
| حقوق: | undefined |
| رقم الانضمام: | edsbas.887503AB |
| قاعدة البيانات: | BASE |
| الوصف غير متاح. |