학술논문
Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.
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article
Author
Tulinius, Mar; Ryan, Monique; Webster, Richard; Castro, Diana; Finkel, Richard; Smith, Andrea; Morgenroth, Lauren; Arrieta, Adrienne; Shimony, Maya; Jaros, Mark; Shale, Phil; McCall, John; Hathout, Yetrib; Nagaraju, Kanneboyina; van den Anker, John; Ward, Leanne; Ahmet, Alexandra; Cornish, Michaelyn; Clemens, Paula; Conklin, Laurie; Damsker, Jesse; Hoffman, Eric; Jusko, William; Mavroudis, Panteleimon; Schwartz, Benjamin; Mengle-Gaw, Laurel; Smith, Edward; Mah, Jean; Guglieri, Michela; Nevo, Yoram; Kuntz, Nancy; McDonald, Craig
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Abstract
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 to