RT Journal Article
T1 Growth parameters in children with achondroplasia: A 7-year, prospective, multinational, observational study.
A1 Savarirayan, Ravi
A1 Irving, Melita
A1 Harmatz, Paul
A1 Delgado, Borja
A1 Wilcox, William R
A1 Philips, John
A1 Owen, Natalie
A1 Bacino, Carlos A
A1 Tofts, Louise
A1 Charrow, Joel
A1 Polgreen, Lynda E
A1 Hoover-Fong, Julie
A1 Arundel, Paul
A1 Ginebreda, Ignacio
A1 Saal, Howard M
A1 Basel, Donald
A1 Font, Rosendo Ullot
A1 Ozono, Keiichi
A1 Bober, Michael B
A1 Cormier-Daire, Valerie
A1 Le Quan Sang, Kim-Hanh
A1 Baujat, Genevieve
A1 Alanay, Yasemin
A1 Rutsch, Frank
A1 Hoernschemeyer, Daniel
A1 Mohnike, Klaus
A1 Mochizuki, Hiroshi
A1 Tajima, Asako
A1 Kotani, Yumiko
A1 Weaver, David D
A1 White, Klane K
A1 Army, Clare
A1 Larrimore, Kevin
A1 Gregg, Keith
A1 Jeha, George
A1 Milligan, Claire
A1 Fisheleva, Elena
A1 Huntsman-Labed, Alice
A1 Day, Jonathan
K1 Achondroplasia
K1 Annualized growth velocity
K1 Anthropometrics
K1 Observational
K1 Pediatrics
AB This study was undertaken to collect baseline growth parameters in children with achondroplasia who might enroll in interventional trials of vosoritide, and to establish a historical control. In this prospective, observational study, participants (≤17 years) underwent a detailed medical history and physical examination and were followed every 3 months until they finished participating in the study by enrolling in an interventional trial or withdrawing. A total of 363 children were enrolled (28 centers, 8 countries). Mean (SD) follow up was 20.4 (15.0) months. In participants  This study represents one of the largest datasets of prospectively collected medical and longitudinal growth data in children with achondroplasia. It serves as a robust historical control to measure therapeutic interventions against and to further delineate the natural history of this condition.
YR 2022
FD 2022-09-16
LK http://hdl.handle.net/10668/22253
UL http://hdl.handle.net/10668/22253
LA en
DS RISalud
RD Apr 12, 2025