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

Savarirayan, Ravi; Tofts, Louise; Irving, Melita; Wilcox, William; Bacino, Carlos; Hoover-Fong, Julie; Ullot Font, Rosendo; Harmatz, Paul; Rutsch, Frank; Bober, Michael; Polgreen, Lynda; Ginebreda, Ignacio; Mohnike, Klaus; Charrow, Joel; Hoernschemeyer, Daniel; Ozono, Keiichi; Alanay, YASEMİN; Arundel, Paul; Kagami, Shoji; Yasui, Natsuo; White, Klane; Saal, Howard; Leiva-Gea, Antonio; Luna-Gonzalez, Felipe; Mochizuki, Hiroshi; Basel, Donald; Porco, Dania; Jayaram, Kala; Fisheleva, Elena; Huntsman-Labed, Alice; Day, Jonathan

doi:10.1016/s0140-6736(20)31541-5

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 (SCI-Expanded, Scopus)

Publication Type: Article / Article
Volume: 396 Issue: 10252
Publication Date: 2020
Doi Number: 10.1016/s0140-6736(20)31541-5
Journal Name: LANCET
Journal Indexes: 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
Page Numbers: pp.684-692
Acibadem Mehmet Ali Aydinlar University Affiliated: Yes

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.