TY  - JOUR
AU  - Savarirayan, Ravi
AU  - Tofts, Louise
AU  - Irving, Melita
AU  - Wilcox, William R
AU  - Bacino, Carlos A
AU  - Hoover-Fong, Julie
AU  - Font, Rosendo Ullot
AU  - Harmatz, Paul
AU  - Rutsch, Frank
AU  - Bober, Michael B
AU  - Polgreen, Lynda E
AU  - Ginebreda, Ignacio
AU  - Mohnike, Klaus
AU  - Charrow, Joel
AU  - Hoernschemeyer, Daniel
AU  - Ozono, Keiichi
AU  - Alanay, Yasemin
AU  - Arundel, Paul
AU  - Kotani, Yumiko
AU  - Yasui, Natsuo
AU  - White, Klane K
AU  - Saal, Howard M
AU  - Leiva-Gea, Antonio
AU  - Luna-González, Felipe
AU  - Mochizuki, Hiroshi
AU  - Basel, Donald
AU  - Porco, Dania M
AU  - Jayaram, Kala
AU  - Fisheleva, Elena
AU  - Huntsman-Labed, Alice
AU  - Day, Jonathan R S
PY  - 2021
DO  - 10.1038/s41436-021-01287-7
UR  - https://hdl.handle.net/10668/27064
T2  - Genetics in medicine : official journal of the American College of Medical Genetics
AB  - Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in...
LA  - en
KW  - Achondroplasia
KW  - Child
KW  - Double-Blind Method
KW  - Humans
KW  - Natriuretic Peptide, C-Type
KW  - Treatment Outcome
TI  - Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.
TY  - research article
VL  - 23
ER  -