Clinical trial highlights

Vamorolone is a first-in-class steroidal anti-inflammatory drug with novel structure/activity relationships with glucocorticoid and mineralocorticoid receptor targets compared to deflazacort or prednisone.   Published open-label dose-finding studies (0.25–6.0 mg/kg/day) in DMD showed significant motor function improvement over 24 weeks for 2.0 and 6.0 mg/kg/day dose groups (n=48; age 4 to

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: E. Hoffman, U. Dang, P. Clemens, H. Gordish-Dressman, B. Schwartz, L. Mengle-Gaw, M. Leinonen, E. Smith, D. Castro, N. Kuntz, R. Finkel, M. Tulinius, Y. Nevo, M. Ryan, R. Webster, J. van den Anker, L. Ward, J. Damsker, C. McDonald, M. Guglieri, J. Mah Source Type: research