TY  - JOUR
AU  - Savarirayan, Ravi
AU  - De Bergua, Josep Maria
AU  - Arundel, Paul
AU  - McDevitt, Helen
AU  - Cormier-Daire, Valerie
AU  - Saraff, Vrinda
AU  - Skae, Mars
AU  - Delgado, Borja
AU  - Leiva-Gea, Antonio
AU  - Santos-Simarro, Fernando
AU  - Salles, Jean Pierre
AU  - Nicolino, Marc
AU  - Rossi, Massimiliano
AU  - Kannu, Peter
AU  - Bober, Michael B
AU  - Phillips, John
AU  - Saal, Howard
AU  - Harmatz, Paul
AU  - Burren, Christine
AU  - Gotway, Garrett
AU  - Cho, Terry
AU  - Muslimova, Elena
AU  - Weng, Richard
AU  - Rogoff, Daniela
AU  - Hoover-Fong, Julie
AU  - Irving, Melita
PY  - 2022
DO  - 10.1177/1759720X221084848
SN  - 1759-720X
UR  - http://hdl.handle.net/10668/20211
T2  - Therapeutic advances in musculoskeletal disease
AB  - Achondroplasia is the most common short-limbed skeletal dysplasia resulting from gain-of-function pathogenic variants in fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Most treatment options are...
LA  - en
KW  - achondroplasia
KW  - clinical trial
KW  - fibroblast growth factor receptor 3
KW  - infigratinib
TI  - Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies.
TY  - research article
VL  - 14
ER  -