Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy and results from mutations in the dystrophin gene. Currently no treatment is available for this devastating condition. Standards of care have been improving and are spreading world-wide, prolonging survival and increasing quality of life, however management of symptoms and complications remains the only interventions that can currently be offered to patients. New and promising experimental strategies to address DMD have been developed over the last few years and some of them are in or are approaching clinical trials. Different therapeutic options are being investigated, ranging from mutation-specific treatments, including nonsense codon suppressors and exon skipping, to gene therapy using viral and nonviral vectors and cell-based approaches. Here we review the current status of molecular investigational treatments in DMD, with a particular focus on those compounds and strategies that are currently in clinical trials or are likely to approach clinical application in the near future.
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