Our Mission

About Cure GM1

The Cure GM1 Foundation’s mission is to fund research that benefits all individuals and families affected by GM1 gangliosidosis. Founded by parents of children with GM1, the Foundation was created with a shared determination to accelerate research, advance treatment development, and ultimately save the lives of those affected by this devastating disease.

The Cure GM1 Foundation is dedicated to directly funding research toward effective treatments and a cure for GM1 gangliosidosis, a rare lysosomal storage disease that progressively damages the brain and spinal cord. GM1 is a severe, degenerative condition associated with a broad and debilitating range of symptoms and complications. For those with the most severe form of the disease, more than half do not survive beyond their fifth birthday.

Cure GM1 is the only 501(c)(3) nonprofit organization entirely dedicated to GM1 gangliosidosis research and drug development. Through strategic research funding, scientific collaboration, and community engagement, the Foundation works to accelerate meaningful progress for everyone affected by GM1.

The Foundation has hosted GM1 research symposiums as well as community and family conferences, bringing together researchers, clinicians, industry leaders, advocates, and families from around the world. These gatherings strengthen collaboration, build community, and help drive momentum toward new treatments and, ultimately, a cure.

To learn more about the GM1 research landscape, recent advancements, and research projects supported or initiated by the Cure GM1 Foundation, please visit our Advancements and Research page.

You can read more about us in our shareable Cure GM1 Foundation PDF.

The Internal Revenue Service approved the foundation’s 501(c)(3) status on April 15th, 2015. With the generous support of GM1 families, those affected by GM1, and the support of the world, we believe a cure can be found. 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 an end to conditions like it, and innocent children who have the incredible misfortune of receiving disease-causing genes can finally have a chance at life.

  • Garcia Hong Law