Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial

@article{Savarirayan2020OncedailySV,
  title={Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial},
  author={Ravi Savarirayan and Louise Tofts and Melita D. Irving and William Wilcox and Carlos A. Bacino and Julie E Hoover-Fong and Rosendo Ullot Font and Paul R. Harmatz and Frank Rutsch and Michael B. Bober and Lynda E Polgreen and Ignacio Ginebreda and Klaus Mohnike and Joel Charrow and Daniel G. Hoernschemeyer and Keiichi Ozono and Yasemin Alanay and Paul Arundel and Shoji Kagami and Natsuo Yasui and Klane K White and Howard M. Saal and Antonio Leiva-Gea and Felipe Luna-Gonz{\'a}lez and Hiroshi Mochizuki and Donald G Basel and Dania M Porco and Kala Jayaram and Elena Fisheleva and Alice Huntsman-Labed and Jonathan Day},
  journal={The Lancet},
  year={2020},
  volume={396},
  pages={684-692}
}
BACKGROUND There are no effective therapies for achondroplasia. An open-label study suggested that vosoritide administration might increase growth velocity in children with achondroplasia. This phase 3 trial was designed to further assess these preliminary findings. METHODS This randomised, double-blind, phase 3, placebo-controlled, multicentre trial compared once-daily subcutaneous administration of vosoritide with placebo in children with achondroplasia. The trial was done in hospitals at… Expand

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