About

about

The Cure GM1 Foundation is dedicated to hope and to directly funding research for a cure for GM1 Gangliosidosis, a lysosomal storage disease that attacks the brain and spinal cord and is always fatal in children.  GM1 is a progressive and degenerative condition with an extremely broad and debilitating array of symptoms and complications.  This nonprofit organization was founded by parents of children who suffer from GM1 who seek to save the lives of all those who suffer from this wretched condition.

The Internal Revenue Service in the IRS approved this foundation’s 501(c)(3) status on April 15th, 2015.  All donations made to the initial campaigns through GoFundMe are not tax exempt as those donations were considered personal gifts.

With the generous support of GM1 families, those affected by GM1, and the support of the world, we believe a a cure can be found.

Research is advancing on multiple fronts globally.  These efforts require financial support.

In particular, our current aim is to support gene therapy research at UMass Medical School and at Auburn University in Alabama. Auburn and UMass are collaborating to bring this research forward. The therapy being developed could cure children in a single treatment.

Learn more about the GM1 research at Auburn University.  Learn more about research at UMass Medical School.

We are also in direct contact with the team of Japanese researchers who are developing pharmacological chaperone/small molecule treatments  that show very promising results.  In cells, the results are dramatic, but human clinical trials have not been initiated.

We must contribute in any way possible to bring these treatments forward.  Every second that goes by,  the world could be closer to an end to GM1 and to an end of conditions like GM1.   Innocent children who have the incredible misfortune of receiving disease-causing genes could have a chance at life.

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