Your support could enable the advancement of Enzyme Replacement Therapy in children diagnosed with GM1.

GM1 Gangliosidosis (GM1) is a rare inherited genetic disorder that primarily affects children. It has many debilitating ramifications, some of which are developmental regression, mobility deterioration, seizures, visual impairment, and neurodegeneration.

GM1 is a fatal disease that currently has no cure or effective treatment. The thousands of diagnosed children and adults who suffer from GM1 face the progressive loss of skills and abilities until death.

Why are we fundraising?

Funding research for ultra rare diseases is incredibly difficult. Because there are so few children suffering from GM1 gangliosidosis, the job of raising money and advocating for research for GM1 falls on our ultra rare disease community.

Currently, the Cure GM1 Foundation is raising money for a potential treatment called Enzyme Replacement Therapy that could exponentially improve outcomes for Iris and hundreds of other children living with GM1 gangliosidosis.

Why Enzyme Replacement Therapy?

Research shows ERT could potentially help patients across all age groups and disease stages by directly supplying a missing enzyme that would enable treatment via AAV gene therapy. ERT represents a critical missing piece in the treatment landscape that could help the majority of GM1 patients, including those who may not benefit from other therapeutic approaches.

ERT has decades of precedent in similar disorders, making the development pathway more established. By developing enzyme replacement therapy for GM1 gangliosidosis, we aim to fill this significant treatment gap and provide hope to families who currently have no options. With your support, we can advance this promising therapy to clinical trials and ultimately work toward an FDA-approved treatment that transforms the lives of children with GM1 gangliosidosis.

Cure GM1 has built a team of advisors and volunteers with decades of prior experience in protein / enzyme replacement therapies. As the project advances, we will also add our regulatory and clinical advisors to the project.

Please donate to the Cure GM1 Foundation to fund the development of potentially life-altering treatments for Iris and other children suffering from GM1 gangliosidosis.

Help fund promising research for children living with GM1 Gangliosidosis