A novel treatment of GM1-gangliosidosis

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

Mots-clé (Anglais)

Life sciences, Pharmaceuticals

Résumé des résultats (Abstract) (Anglais)

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.