MOLÉCULES CHIMIQUES INHIBANT LE MÉCANISME D'ÉPISSAGE POUR TRAITER DES MALADIES RÉSULTANT D'ANOMALIES D'ÉPISSAGE

摘要

The present invention relates to specific compounds; pharmaceutical composition comprising at least one of said compounds; and the use of at least one of said compounds in preparing a drug to treat, in a subject, a genetic disease resulting from at least one splicing anomaly.