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NEW THERAPEUTIC TARGET FOR THE TREATMENT OF MYOTONIC DYSTROPHY
New target for use in the treatment of myotonic dystrophy. The treatment may be based on an oligonucleotide molecule and/or a small molecule that at least partially restores the phenotype of a patient with DM to a non-diseased state. Different molecules have been identified that produce such an effect.
Applications; The main application of the technology is in the pharmaceutical sector, as an active ingredient for the treatment of Myotonic Dystrophy. Competitive advantages: The main advantages provided by the invention are: The identification of the target allows the selection of different drugs. The newly identified target is in the early stages of the pathogenesis pathway. Its reversion to the non-pathogenic state makes it possible that many characteristic phenotypes of the disease can be reversed or even prevented.