There is currently no cure for Duchenne muscular dystrophy, but there are some promising treatments in development, of which antisense-mediated exon skipping is close to clinical application. The results of the first trials have resulted in significant enthusiasm among clinicians, patients, and their parents and attracted widespread attention in both the scientific and lay press. As in many other rare diseases with significant morbidity this fuels the hope that at last an effective therapy might be within our reach. Parents, but also some clinicians, are now seriously considering treating individual patients with antisense oligonucleotides without waiting for proof from well-designed clinical trials that the new therapies are indeed effective and safe.
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