Our main objective is to find and develop treatments to address unmet medical needs. The first target of the company is the investigation of a treatment of myotonic dystrophy Type 1 (DM1), which is an orphan disease affecting more than 900,000 people worldwide (estimated prevalence 1/8,000). Our preclinical research has revealed a new therapeutic target for investigation.
“Investigating antisense RNAs treatments for unmet medical needs”
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