How well does Agamree(Vamorolone) work?

AGAMREE improves motor function in pediatric DMD patients, validated by clinical trials.

Efficacy in Duchenne Muscular Dystrophy

AGAMREE demonstrated efficacy in a 24-week randomized trial (N=121) of ambulatory DMD patients aged 4–7 years. The 6 mg/kg/day dose significantly improved Time to Stand (TTSTAND) velocity (LS mean difference: 0.060 rises/sec vs. placebo, p=0.002) and 6-Minute Walk Test (6MWT) distance (+42 meters, p=0.002). These outcomes reflect preserved muscle strength and mobility. The 2 mg/kg/day dose showed intermediate efficacy but lacked statistical significance for some endpoints. The mechanism remains unclear but involves glucocorticoid receptor-mediated anti-inflammatory effects. No data support use in non-ambulatory or geriatric DMD patients.