Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
Ravi Savarirayan, Louise Tofts, Melita Irving, William R. Wilcox, Carlos A. Bacino, Julie Hoover‐Fong, Rosendo Ullot Font, Paul Harmatz, Frank Rutsch, Michael B. Bober, Lynda E. Polgreen, Ignacio Ginebreda, Klaus Mohnike, Joel Charrow, Daniel G. Hoernschemeyer, Keiichi Ozono, Yasemin Alanay, Paul Arundel, Shoji Kagami, Natsuo Yasui, Klane K. White, Howard M. Saal, Antonio Leiva‐Gea, Felipe Luna-González, Hiroshi Mochizuki, Donald Basel, Dania M Porco, Kala Jayaram, Elena Fisheleva, Alice Huntsman-Labed, Jonathan Day
Topics & Concepts
MedicineAchondroplasiaPlaceboClinical endpointAmbulatoryClinical trialRandomized controlled trialPhysical therapyIntention-to-treat analysisPediatricsInternal medicineAlternative medicinePathologyConnective tissue disorders researchTendon Structure and TreatmentBone and Dental Protein Studies