Our main objective is finding effective treatments to unmet medical needs. The first target of the company will be to investigate anti-microRNAs for the treatment of myotonic dystrophy Type 1 (DM1), which is an orphan disease affecting more than 900.000 people worldwide (estimated prevalence 1/8000). Our research has reveled new therapeutic targets to treat the disease. The target and the mechanism of action of our molecules is new, and no other competitor has developed a similar approach.
“antisense RNAs treatments for genetic disorders”.
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