PURPOSE OF REVIEW: The most encouraging recent advances regarding 
pharmacological agents for treating Duchenne muscular dystrophy (DMD) are 
summarized. Emphasis is given to compounds acting downstream of dystrophin, the 
protein lacking in DMD, on cellular pathways leading to pathological 
consequences. The author highlights the progress that may have the greatest 
potential for clinical use in DMD.
RECENT FINDINGS: Modifying the transcripts of the mutated gene by exon skipping 
has led to expression of shortened dystrophins in DMD patients. Currently, the 
most promising potential drugs are the exon-skipping agents eteplirsen and 
drisapersen. Biglycan and SMTC1100 upregulate utrophin. The steroid receptor 
modulating compounds VBP15 and tamoxifen, and specific antioxidants appear 
promising agents for symptomatic therapy.
SUMMARY: The past 18 months have seen a strong increase in the number of 
exciting reports on novel therapeutic agents for DMD. Exon-skipping agents have 
been fine-tuned to improve tissue delivery and stability. Impressive discoveries 
regarding pathogenic events in cellular signalling have revealed targets that 
were unknown in the context of DMD, thus enabling approaches that limit 
inflammation, fibrosis and necrosis. The targets are nuclear hormone receptors, 
NADPH-oxidases and Ca channels. Inhibition of NF-KB, transforming growth 
factor-alpha (TGF-α) and transforming growth factor-beta (TGF-β)/myostatin 
production or action are also promising routes in counteracting the complex 
pathogenesis of DMD.