Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

RCT (n=16) found that viltolarsen 40 mg/kg & 80 mg/kg were well tolerated with no treatment-emergent adverse events requiring dose changes or cessation of treatment. Compared with matched controls all patients had improvements in time to stand from supine, and time to walk 10m.

Source:

JAMA Neurology