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A Randomized, Double‐Blind, Placebo‐Controlled, Multiple Dose Escalation Study to Evaluate the Safety and Pharmacokinetics of ELX‐02 in Healthy Subjects

ELX‐02 is an investigational compound being developed as a therapy for genetic diseases caused by nonsense mutations such as cystic fibrosis. Structurally, ELX‐02 is an aminoglycoside analogue that induces read‐through of nonsense mutations through interaction with the ribosome, resulting in the pro...

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Detalles Bibliográficos
Autores principales:	Leubitz, Andi, Vanhoutte, Frederic, Hu, Ming‐yi, Porter, Kaela, Gordon, Efrat, Tencer, Kathleen, Campbell, Kathleen, Banks, Kate, Haverty, Tom
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2021
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8451797/ https://www.ncbi.nlm.nih.gov/pubmed/33465285 http://dx.doi.org/10.1002/cpdd.914

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