Frieda’s Story – An Update
How is Frieda doing right now with her symptoms, the progression of the disease, and…
How is Frieda doing right now with her symptoms, the progression of the disease, and…
Batul’s Story Batul passed away from infantile GM1 (Type 1) on March 23, 2026. Below,…
Florence’s Story: An Update Back in 2019, we shared the story of Florence, who was…
Leonor’s Story: An Update Back in 2019, we shared the story of Leonor, who was…
For families living with GM1 or GM2 gangliosidosis, every day is a marathon. These rare, progressive neurological diseases, caused by enzyme deficiencies that lead to the toxic buildup of lipids in the brain and nervous system, have no approved treatments and no cure. But beyond the devastating impact on those diagnosed, a growing body of research is shining a necessary light on another group of people profoundly affected: the caregivers.
As the year comes to an end, we remain focused on creating connection by keeping our rare disease community informed of advocacy opportunities, research news and community updates. Join us in learning more about our efforts to push for a cure for GM1 gangliosidosis.
Reece’s Story, By His Mother A GM1 Type 2 Story(Published April 1, 2026) “Reece, we…
Receiving a diagnosis for a child with a disease like GM1 can be overwhelming and life-changing. Parents often experience grief, anxiety, and a sense of loss as their expectations for parenthood and their child’s future change.
Could a service dog benefit your child living with GM1 gangliosidosis? At the 2023 Cure GM1 Community Conference, Brad Wilson, a full-time police officer and parent to Tyler, a child living with Angelman Syndrome, shared the ways a “skilled companion” service dog from Canine Companions benefited his son.