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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Publicat a:Neurology
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Lippincott Williams & Wilkins 2019
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Article
Accés en línia: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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