Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study

Author:

Savarirayan RaviORCID,Tofts Louise,Irving Melita,Wilcox William R.,Bacino Carlos A.,Hoover-Fong Julie,Font Rosendo Ullot,Harmatz Paul,Rutsch Frank,Bober Michael B.,Polgreen Lynda E.,Ginebreda Ignacio,Mohnike Klaus,Charrow Joel,Hoernschemeyer Daniel,Ozono Keiichi,Alanay Yasemin,Arundel Paul,Kotani Yumiko,Yasui Natsuo,White Klane K.,Saal Howard M.,Leiva-Gea Antonio,Luna-González Felipe,Mochizuki Hiroshi,Basel Donald,Porco Dania M.,Jayaram Kala,Fisheleva Elena,Huntsman-Labed Alice,Day Jonathan R.S.

Publisher

Elsevier BV

Subject

Genetics (clinical)

Reference8 articles.

1. Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia;Shiang,1994

2. Achondroplasia;Horton,2007

3. Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia;Lorget,2012

4. Overexpression of CNP in chondrocytes rescues achondroplasia through a MAPK-dependent pathway;Yasoda,2004

5. C-type natriuretic peptide analogue therapy in children with achondroplasia;Savarirayan,2019

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