Jong-Hee Chae

TL;DR: Those who received nusinersen were more likely to be alive and have improvements in motor function than those in the control group and infants with a shorter disease duration at screening wereMore likely than those with a longer disease duration to benefit from nusineren.

TL;DR: Ataluren was generally well tolerated and most treatment-emergent adverse events were mild to moderate in severity, and there was a significant effect of ataluren in the prespecified subgroup of patients in the intention-to-treat population.

TL;DR: In this paper, the authors compare the performance of deflazacort and prednisone/prednisolone in providing clinically meaningful delays in loss of physical milestones in patients with nonsense mutation Duchenne muscular dystrophy.