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Once-weekly TransCon CNP (navepegritide) in children with achondroplasia (ACcomplisH): a phase 2, multicentre, randomised, double-blind, placebo-controlled, dose-escalation trial

BACKGROUND: TransCon CNP (navepegritide) is an investigational prodrug of C-type natriuretic peptide (CNP) designed to allow for continuous CNP exposure with once-weekly dosing. This 52-week phase 2 (ACcomplisH) trial assessed the safety and efficacy of TransCon CNP in children with achondroplasia....

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Detalles Bibliográficos
Autores principales: Savarirayan, Ravi, Hoernschemeyer, Daniel G., Ljungberg, Merete, Zarate, Yuri A., Bacino, Carlos A., Bober, Michael B., Legare, Janet M., Högler, Wolfgang, Quattrin, Teresa, Abuzzahab, M. Jennifer, Hofman, Paul L., White, Klane K., Ma, Nina S., Schnabel, Dirk, Sousa, Sérgio B., Mao, Meng, Smith, Alden, Chakraborty, Mukta, Giwa, Adebola, Winding, Bent, Volck, Birgitte, Shu, Aimee D., McDonnell, Ciara
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10562841/
https://www.ncbi.nlm.nih.gov/pubmed/37823031
http://dx.doi.org/10.1016/j.eclinm.2023.102258