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Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

OBJECTIVE: To study vamorolone, a first-in-class steroidal anti-inflammatory drug, in Duchenne muscular dystrophy (DMD). METHODS: An open-label, multiple-ascending-dose study of vamorolone was conducted in 48 boys with DMD (age 4–<7 years, steroid-naive). Dose levels were 0.25, 0.75, 2.0, and 6.0...

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Detalhes bibliográficos
Publicado no:Neurology
Main Authors: Hoffman, Eric P., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Castro, Diana, Mah, Jean K., McDonald, Craig M., Kuntz, Nancy L., Finkel, Richard S., Guglieri, Michela, Bushby, Katharine, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Siener, Catherine, Jaros, Mark, Shale, Phil, McCall, John M., Nagaraju, Kanneboyina, van den Anker, John, Conklin, Laurie S., Cnaan, Avital, Gordish-Dressman, Heather, Damsker, Jesse M., Clemens, Paula R.
Formato: Artigo
Idioma:Inglês
Publicado em: Lippincott Williams & Wilkins 2019
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7011869/
https://ncbi.nlm.nih.gov/pubmed/31451516
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1212/WNL.0000000000008168
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