Brain and nerve = Shinkei kenkyū no shinpo
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Duchenne muscular dystrophy (DMD) is the most common form of inherited muscle disease and is characterized by progressive muscle wasting ultimately resulting in death of the patients in their twenties. DMD is characterized by a deficiency of the muscle dystrophin as a result of mutations in the dystrophin gene. Currently, no effective treatment for DMD is available. ⋯ Induction of the read-through effect using gentamycin or PTC124 is expected to produce dystrophin in DMD patients with nonsense mutation. The treatment with PTC124 is currently under clinical trial. In this review, these treatments strategies have been summarized.