The EPNS Scientific and Research Committee together with the Young EPNS (YEPNS) are hosting the EPNS Journal Club in the shape of interactive webinars discussing selected articles of key interest to Child Neurologists on the 4th Wednesday of every other month at 17:00h CET.
Duchenne muscular dystrophy (DMD) is an X-linked severe muscular disease, in which corticosteroid anti-inflammatory drugs are used to delay loss of motor function. The side effects of this medication can preclude a long-term adherence to the therapeutic plan. Vamolorone, a newly designed steroidal anti-inflammatory drug, intends to retain the beneficial anti-inflammatory and muscle strengthening aspects of corticosteroids, while decreasing some of the undesirable side effects. We will discuss the results of a double-blind, 24-week multicenter clinical trial of 121 corticosteroid-naïve DMD boys, randomly assigned to a placebo, prednisolone (0.75 mg/kg) or vamolorone (2 or 6 mg/kg) group. Main outcomes included efficacy (motor outcomes) and safety.
ARTICLE: RCT on Vamorolone vs placebo and prednisone in Duchenne (Guglieri M, Clemens PR, Perlman SJ et al. Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Neurol. 2022 Oct 1;79(10):1005-1014. doi: 10.1001/jamaneurol.2022.2480.