Dorphan Small Molecule Research

Cure GM1 most’s recent grant was for a drug repurposing project and mouse study to study a small molecule drug in collaboration with a small biotech company called Dorphan located in Switzerland.

UC Davis Lentiviral Gene Therapy Research

Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using stem cells.  The research is being conducted at University of California Davis.

Auburn/UMass Research AAV9 IV Gene Therapy

Cure GM1 funded pre-clinical studies and IND-enabling studies for AAV9 intravenous GM1 gene therapy being conducted by investigators at UMass Medical School and Auburn Univesity.  Please see this video on Auburn’s research and Auburn’s web site.

Cyclodextrin GM1 Pilot Study

GM1 Cyclodextrin Pilot Study Initiated


Additional GM1 Research

Syner-G Clinical Study

There is clinical study being conducted at University of Minnesota where the ketogenic diet is combined with a medicine called miglustat/Zavesca.  Unfortunately, this regimen is not considered a cure.  The regimen may only slow the course of GM1 for certain patients.  It is thought to possibly be effective for certain genetic mutations or only Juvenile patients or those with residual enzyme activity.  Please refer to the description on the clinical trials web site.