Abstract: Compound for treatment of myotonic dystrophy type The present invention relates to a compound, or a salt thereof, and a pharmaceutical composition comprising said compound, or a salt thereof, for use in the prevention and/or treatment of myotonic dystrophy type 1 in a patient, in particular by reducing the levels of autophagy and apoptosis that are elevated in myotonic muscular dystrophy type 1.

Abstract: The present invention refers to phenanthrene derivatives for use as medicaments, mainly in the prevention and/or treatment of Myotonic Dystrophy Type 1, Huntington's Disease Like 2, Spinocerebellar Ataxia Type 8, Myotonic Dystrophy Type 2, Spinocerebellar Ataxia Type 3, Fragile-X-Associated Tremor/Ataxia Syndrome, Frontotemporal Degeneration/Amyotrophic Lateral Sclerosis and Spinocerebellar Ataxia Type 31. In a preferred embodiment, phenanthrene derivatives of the invention are also used as antimyotonic agents. Therefore, this invention may be included either in the whole pharmaceutical or medical field.

Abstract: Compounds to be used in the treatment of diseases based on the expression of toxic transcripts. The present invention relates to peptide molecules, specifically hexapeptides, designed for the prevention and/or treatment of diseases the etiology whereof is based on the presence of toxic transcripts that comprise CUG, CCUG, CGG, CAG and AAG repeats, preferably: DM1 SCA8, DM2, FXTAS, HD and FA.