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., ...Daha Fazla

LANCET, cilt.396, sa.10252, ss.684-692, 2020 (SCI-Expanded) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 396 Sayı: 10252
  • Basım Tarihi: 2020
  • Doi Numarası: 10.1016/s0140-6736(20)31541-5
  • Dergi Adı: LANCET
  • Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, PASCAL, Agricultural & Environmental Science Database, Aquatic Science & Fisheries Abstracts (ASFA), BIOSIS, CAB Abstracts, EMBASE, Food Science & Technology Abstracts, MEDLINE, Psycinfo, Veterinary Science Database
  • Sayfa Sayıları: ss.684-692
  • Acıbadem Mehmet Ali Aydınlar Üniversitesi Adresli: Evet

Özet

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.