Results of North Star ambulatory assessments in the phase 3 ataluren confirmatory trial in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD)

A Potential New Therapeutic Approach to DMD: PKC Theta Inhibition

“…Gene replacement therapy constitutes the best hope for treating DMD. However, gene therapies are still under development ( Muntoni et al, 2016 ) and the only medication so far able to retard the progression of DMD are corticoids. ( Matthews et al, 2016 )…”