A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

BACKGROUND: Edasalonexent (CAT-1004) is an orally-administered novel small molecule drug designed to inhibit NF-κB and potentially reduce inflammation and fibrosis to improve muscle function and thereby slow disease progression and muscle decline in Duchenne muscular dystrophy (DMD). OBJECTIVE: This...

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Detalles Bibliográficos
Autores principales: Finkel, Richard S., McDonald, Craig M., Lee Sweeney, H., Finanger, Erika, Neil Knierbein, Erin, Wagner, Kathryn R., Mathews, Katherine D., Marks, Warren, Statland, Jeffrey, Nance, Jessica, McMillan, Hugh J., McCullagh, Gary, Tian, Cuixia, Ryan, Monique M., O’Rourke, Declan, Müller-Felber, Wolfgang, Tulinius, Mar, Burnette, W. Bryan, Nguyen, Cam-Tu, Vijayakumar, Kayal, Johannsen, Jessika, Phan, Han C., Eagle, Michelle, MacDougall, James, Mancini, Maria, Donovan, Joanne M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2021
Materias:
Research Report
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8543277/
https://www.ncbi.nlm.nih.gov/pubmed/34120912
http://dx.doi.org/10.3233/JND-210689

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