PSAT106 Infigratinib in Children with Achondroplasia: Design of the PROPEL, PROPEL2 and PROPEL OLE Studies

BACKGROUND: Achondroplasia (ACH), the most common non-lethal form of skeletal dysplasia, is characterized by defective endochondral ossification resulting from gain-of-function mutations in the fibroblast growth factor receptor 3 (FGFR3) gene, a negative regulator of endochondral bone formation. Inf...

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Detalles Bibliográficos
Autores principales: Muslimova, Elena, De Bergua, Josep Maria, Savarirayan, Ravi, Arundel, Paul, McDevitt, Helen, Cormier-Daire, Valerie, Saraff, Vrinda, Skae, Mars, Delgado, Borja, Leiva-Gea, Antonio, Santos-Simarro, Fernando, Pierre Salles, Jean, Nicolino, Marc, Rossi, Massimiliano, Kannu, Peter, Bober, Michael B, Phillips III, John, Saal, Howard, Harmatz, Paul, Burren, Christine, Gotway, Garrett, Cho, Terry, Weng, Richard, Rogoff, Daniela, Hoover-Fong, Julie, Irving, Melita
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Pediatric Endocrinology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9627626/
http://dx.doi.org/10.1210/jendso/bvac150.1311

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