A novel treatment of GM1-gangliosidosis (Innolink: 102.301 IP-LS)

• Unité de recherche: INNOSUISSE
• Numéro de projet: 62242.1 IP-LS
• Titre du projet anglais: A novel treatment of GM1-gangliosidosis (Innolink: 102.301 IP-LS)

Description succincte (Allemand)

A novel treatment of GM1-gangliosidosis (Innolink: 102.301 IP-LS)

Description succincte (Anglais)

A novel treatment of GM1-gangliosidosis

Résumé des résultats (Abstract) (Allemand)

Dorphan has shown that Aloxistatin has a therapeutic activity in cells from GM1-gangliosidosis patients. Aloxistatin is a poor drug and has a low brain penetration. We have discovered molecules derived from it and we want to select the best out of them to offer a valuable therapy to the patients.