Small molecule drug for the treatment of myotonic dystrophy

• Unité de recherche: INNOSUISSE
• Numéro de projet: 102.272 IP-LS
• Titre du projet anglais: Small molecule drug for the treatment of myotonic dystrophy

Mots-clé (Anglais)

Life sciences, Human medicine

Résumé des résultats (Abstract) (Anglais)

The aim of the project is to obtain pre-clinical development of two patent-protected small molecule drug candidates in animal models for the treatment of myotonic dystrophy. The outcome will generate IND-enabling data for further validation in non-human primates and eventual clinical phase entry.