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Join Our Race To Save Children
GM1 gangliosidosis was first identified in 1968, yet for decades it lacked the research and advocacy it desperately needed. Children diagnosed with GM1 face a devastating prognosis, and their families have too often been left without hope. Cure GM1 is a 501(c)(3) nonprofit with one focus: GM1. Every dollar we raise funds promising research, accelerates drug development, and amplifies the voices of patients and families who deserve better.
~$6.1M
Cure GM1 has raised significant funding and driven meaningful progress, but more funding is still required.
5
Since Cure GM1 was founded, there have been 5 clinical trials, whereas prior to Cure GM1’s existence, there were none.
95%
95% of the 10,000+ rare diseases have no treatments.
GM1 Gangliosidosis Community Stories
Take action and join us
Participate and Take Action
Join the GM1 Census and Patient Registry
GM1 families have the power to change the understanding of GM1. The GM1 census is a critical tool to deepen the understanding of GM1, mapping the diagnostic journey, identifying common symptoms and demonstrating to biotech companies and interested parties that our community is research-ready. Join now.
Annual International GM1 Community Conference
Our Fall annual conference is an extremely valuable resource to GM1 families and to researchers and biotechs working on GM1. The presentations provide incredibly valuable information regarding GM1, clinical trials, caregiving tips and more.
Do It for GM1
Do It For GM1 is a movement bringing us closer to a world where children with GM1 can live longer, healthier lives. Choose an activity, rally your community, and help us accelerate critical research for GM1 families.
Rare Disease Day
Raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and caregivers.
International GM1 Gangliosidosis Awareness Day
Organized by Cure GM1 in collaboration with multiple patient advocacy groups, International GM1 Gangliosidosis Awareness Day is a worldwide movement to advance advocacy, research funding, and drug development to improve and save lives.
Take Meaningful Action and Help Advance our Mission
Our work depends on volunteers and active participation in GM1 research, advocacy, and fundraising. Watch a video about our critically important work.
Helpful resources
Help Build Resources, Tools, knowledge and Community
Donations, volunteers, and our global community fuel our critically important work to help develop and advance possible treatments.
Our Blog
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The Hidden Weight of Rare Disease: What Research Reveals About the Caregiving Burden on GM1 and GM2 Caregivers
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.
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How to Handle More Than You Can Handle: Navigating the Emotional Experience of Caring for and Raising Disabled Children with High-Support Needs
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.
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Canine Companions and Their Impact on Special Needs Families
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.
GM1 and rare disease community
collaboration, advocacy and community are critical
Cure GM1 has been instrumental in helping build community and to structure meaningful projects and collaboration amongst stakeholders. Some highlights include the organization of a natural history data sharing effort, the development of a newborn screening assay, and interactions with the U.S. Food and Drug Adminstration, FDA.
Jenny Bragg,
GM1 parent
“If I could change Clara’s future, I would. Nevertheless, I am forever grateful for the impact she has had on my life as well as countless others.”
Kylie Harrison,
Board Member
“People are telling us there’s nothing we can do for our daughters, we’re not accepting that. There is something we can do and that is to raise awareness and raise funding for them and for others.
Christine Waggoner
Founder
“Cure GM1 is laser-focused on creating a legacy of hope and change by navigating the many challenges of ultra-rare disease drug development and research.”
Emil Kakkis, Advisor
“I believe that GM1 is at a place where it can get treated. But you have to be vigilant and fight for support to ensure that the right things get done and that treatments become available.”
SAFE + EASY DONATIONS and fundraising
Ways to Donate and Fundraise
Download our fundraising manual. Read our refund policy.
Contact us for help
Email info@curegm1.org
We’re Changing
GM1 Research Globally
Our work and advocacy contributes to brings increased awareness, scientific knowledge, and financial investment.
11+
YEARS OF ADVOCACY
5
CLINICAL TRIALS TO DATE
11
SCIENTIFIC PUBLICATIONS
2
PUBLICLY AVAILABLE MOUSE MODELS