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.


JAMA Neurology