ANTISENSE RNA-THERAPEUTICS IN MYOTONIC DYSTROPHY - Banco de Patentes

Myotonic Dystrophy (DM, overall prevalence of 1/8000) is a highly disabling multisystemic disease with symptoms involving the nervous system, the heart, and the skeletal musculature. In its most common form, onset of symptoms occurs during adolescence and affected individuals have a significantly shortened lifespan of 48-55 years. Currently, DM is an unmet medical need. Researchers from INCLIVA has discovered that the inhibition of a microRNAs by antisense molecules can be therapeutic for the treatment of DM. The project is highly translational and our broad experience, preliminary data, collaboration with leading international researchers in DM1, and the interest by biotech companies, offer additional guaranties of success