Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug

We report a first-in-patient study of vamorolone, a first-in-class dissociative steroidal anti-inflammatory drug, in Duchenne muscular dystrophy. This 2-week, open-label Phase IIa multiple ascending dose study (0.25, 0.75, 2.0, and 6.0 mg/kg/day) enrolled 48 boys with Duchenne muscular dystrophy (4...

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Vydáno v:Pharmacol Res
Hlavní autoři: Conklin, Laurie S., Damsker, Jesse M., Hoffman, Eric P., Jusko, William J., Mavroudis, Panteleimon D., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Mah, Jean K., Guglieri, Michela, Nevo, Yoram, Kuntz, Nancy, McDonald, Craig M., Tulinius, Mar, Ryan, Monique M., Webster, Richard, Castro, Diana, Finkel, Richard S., Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Jaros, Mark, Shale, Phil, McCall, John M., Hathout, Yetrib, Nagaraju, Kanneboyina, van den Anker, John, Ward, Leanne M., Ahmet, Alexandra, Cornish, Michaelyn R., Clemens, Paula R.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2018
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6218284/
https://ncbi.nlm.nih.gov/pubmed/30219580
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.phrs.2018.09.007
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