Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-κB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

BACKGROUND: Edasalonexent is an orally administered small molecule designed to inhibit NF-κB, which is activated from infancy in Duchenne muscular dystrophy and is central to causing muscle damage and preventing muscle regeneration. OBJECTIVE: Evaluate the safety, tolerability, pharmacokinetics and...

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Detalles Bibliográficos
Autores principales: Finanger, Erika, Vandenborne, Krista, Finkel, Richard S., Lee Sweeney, H., Tennekoon, Gihan, Yum, Sabrina, Mancini, Maria, Bista, Pradeep, Nichols, Andrew, Liu, Hanlan, Fretzen, Angelika, Donovan, Joanne M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2019
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6398836/
https://www.ncbi.nlm.nih.gov/pubmed/30452422
http://dx.doi.org/10.3233/JND-180341

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