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


Savarirayan R., Tofts L., Irving M., Wilcox W., Bacino C. A. , Hoover-Fong J., ...More

LANCET, vol.396, no.10252, pp.684-692, 2020 (Journal Indexed in SCI) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 396 Issue: 10252
  • Publication Date: 2020
  • Doi Number: 10.1016/s0140-6736(20)31541-5
  • Title of Journal : LANCET
  • Page Numbers: pp.684-692

Abstract

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.