1. SAT-LB18 A Randomized Controlled Trial of Vosoritide in Children With Achondroplasia
- Author
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Klane K. White, Felipe Luna-Gonzáles, Antonio Leiva-Gea, Lynda E. Polgreen, Rosendo Ullot Font, Alice Huntsman-Labed, Donald Basel, Natsuo Yasui, Dania M Porco, Jonathan Day, Elena Fisheleva, Hiroshi Mochizuki, Melita Irving, Paul Harmatz, Keiichi Ozono, Daniel Hoernschmeyer, Yasemin Alanay, Julie Hoover-Fong, Joel Charrow, Kala Jayaram, Howard M. Saal, Frank Rutsch, Carlos A. Bacino, Klaus Mohnike, Ignacio Ginebreda, Paul Arundel, William R. Wilcox, Michael B. Bober, Ravi Savarirayan, Louise Tofts, and Shoji Kagami
- Subjects
Pediatrics ,medicine.medical_specialty ,business.industry ,Endocrinology, Diabetes and Metabolism ,medicine.disease ,law.invention ,Randomized controlled trial ,Pediatric Endocrinology ,law ,medicine ,Pediatric Growth and Adrenal Disorders ,Achondroplasia ,business ,AcademicSubjects/MED00250 ,Vosoritide - Abstract
Background: Achondroplasia is a disorder caused by specific mutations in the gene encoding the fibroblast growth factor receptor 3 (FGFR3) protein. Open-label, phase 2 trials in children with achondroplasia showed that administration of vosoritide, an analogue of C-natriuretic peptide, resulted in sustained increases in annualized growth velocity. Methods: This international, randomized, double-blind, phase 3 trial compared once-daily subcutaneous administration of vosoritide, at a dose of 15 μg per kg of body weight, with placebo in children with achondroplasia aged 5 to
- Published
- 2020