Rationale, design, and methods of a randomized, controlled, open-label clinical trial with open-label extension to investigate the safety of vosoritide in infants, and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery

Achondroplasia causes narrowing of the foramen magnum and the spinal canal leading to increased mortality due to cervicomedullary compression in infants and significant morbidity due to spinal stenosis later in adulthood. Vosoritide is a C-natriuretic peptide analogue that has been shown to improve...

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Detalles Bibliográficos
Autores principales: Savarirayan, Ravi, Irving, Melita, Maixner, Wirginia, Thompson, Dominic, Offiah, Amaka C, Connolly, Daniel JA, Raghavan, Ashok, Powell, James, Kronhardt, Marcin, Jeha, George, Ghani, Sajda, Fisheleva, Elena, Day, Jonathan RS
Formato: Online Artículo Texto
Lenguaje:English
Publicado: SAGE Publications 2021
Materias:
Short Reports
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10395166/
https://www.ncbi.nlm.nih.gov/pubmed/33761804
http://dx.doi.org/10.1177/00368504211003782

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10395166/
https://www.ncbi.nlm.nih.gov/pubmed/33761804
http://dx.doi.org/10.1177/00368504211003782

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