In contrast to drisapersen (Kyndrisa, BioMarin Pharmaceutical), eteplirsen (Exondys, Sarepta Therapeutics) received accelerated approval in 2016 by the FDA based on a slight increase in dystrophin levels, 0.4% and 0.9% of the control level, in 13 and 11 DMD patients after 48 and 180 weeks of treatment, respectively. The gene discussed is DMD; the disease is Duchenne muscular dystrophy.