Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial

IMPORTANCE: Corticosteroidal anti-inflammatory drugs are widely prescribed but long-term use shows adverse effects that detract from patient quality of life. OBJECTIVE: To determine if vamorolone, a structurally unique dissociative steroidal anti-inflammatory drug, is able to retain efficacy while r...

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Detalles Bibliográficos
Autores principales: Guglieri, Michela, Clemens, Paula R., Perlman, Seth J., Smith, Edward C., Horrocks, Iain, Finkel, Richard S., Mah, Jean K., Deconinck, Nicolas, Goemans, Nathalie, Haberlova, Jana, Straub, Volker, Mengle-Gaw, Laurel J., Schwartz, Benjamin D., Harper, Amy D., Shieh, Perry B., De Waele, Liesbeth, Castro, Diana, Yang, Michelle L., Ryan, Monique M., McDonald, Craig M., Tulinius, Mar, Webster, Richard, McMillan, Hugh J., Kuntz, Nancy L., Rao, Vashmi K., Baranello, Giovanni, Spinty, Stefan, Childs, Anne-Marie, Sbrocchi, Annie M., Selby, Kathryn A., Monduy, Migvis, Nevo, Yoram, Vilchez-Padilla, Juan J., Nascimento-Osorio, Andres, Niks, Erik H., de Groot, Imelda J.M., Katsalouli, Marina, James, Meredith K., van den Anker, Johannes, Damsker, Jesse M., Ahmet, Alexandra, Ward, Leanne M., Jaros, Mark, Shale, Phil, Dang, Utkarsh J., Hoffman, Eric P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Medical Association 2022
Materias:
Original Investigation
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9425287/
https://www.ncbi.nlm.nih.gov/pubmed/36036925
http://dx.doi.org/10.1001/jamaneurol.2022.2480

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