Every single second matters
Iris was born healthy. She is 18 now. GM1 has taken almost everything.
Children with GM1 are born healthy. Then, month by month, the disease takes their movement, their speech, and their ability to swallow. GM1 ultimately causes death. There is still no approved treatment. For the first time, there are treatments in trials. They came too late for Iris. Letโs work together to ensure there is a brighter future and chances at life for all those suffering due to GM1.
Approved treatments for GM1. There is still nothing a doctor can prescribe, and 95% of rare diseases are in the same position.
Since Cure GM1 was founded, there have been 5 clinical trials, whereas prior to Cure GM1’s existence, there were none.
95% of the 10,000+ rare diseases have no treatments.
Candid 2025 Gold Seal | EIN 47-3154419
Your employer may match this gift.
Fund the science that reaches them
Cure GM1 Foundation is a 501(c)(3), EIN 47-3154419. Donations are tax deductible in the USA and we will send a receipt for your records.
Your donation can be spent more than once. We are building a venture philanthropy model, so that if research we funded leads to an approved treatment, a share of the proceeds comes back to Cure GM1 and funds the next project.
Give monthly, become a Cure GM1 Champion
Ongoing, recurring support that lets us plan and commit to research. Select โMonthlyโ in the form, or start a monthly gift โ
Give in memory or tribute
Honor a loved one, mark a milestone, or ask for gifts in lieu of flowers. We will notify the family if you wish. Make a tribute gift โ
Planned and legacy giving
Include Cure GM1 in your will or estate plan. A bequest extends hope for years. Explore planned giving โ
Other ways to give
PayPal Giving Fund, fee free, 100% reaches us, no PayPal account required. Bank transfer or crypto through Every.org. Or mail a check payable to Cure GM1 Foundation, PO Box 6890, Albany, CA 94706.
Where your money actually goes
A drug company will pay for the trial. Nobody will pay for what makes the trial possible.
Before any company will spend $100 million testing a treatment for GM1, someone has to prove the disease can be measured. That means natural history data, biomarkers, patient registries, and animal models. None of it produces a product, so no company funds it. And no trial can be designed without it.
That groundwork is what your donation buys. Cure GM1 has raised $6.2M over eleven years. In that time GM1 went from zero clinical trials to five, drawing industry and government investment that had nowhere to land before the foundational work existed.
GM1 Matrix Patient Portal
Please join. Weโre building natural history and real world data sets. Data support trial design and comparator arms. Without the necessary data, regulators recommend placebo-controlled trials.
Community events
The annual conference brings together families, researchers and biotechs. GM1 Day on May 23 and Rare Disease Day in February build awareness and support, and help raise funds, that pay for this groundwork.
Increased awareness
Awareness helps drive earlier diagnoses. Advocacy keeps GM1 visible to biotechs deciding where to invest, and it shows regulators how severe the unmet need is for GM1.
Support for newborn screening
By the time symptoms appear, irreversible damage is done. Screening is public health infrastructure, and it is what makes early treatment possible at all. In a disease like GM1, early treatment is critical.
Current campaign
Fund the Enzyme Replacement Therapy Project
Enzyme replacement therapy is a reliable and proven path to treatment for diseases like GM1. Carrying it forward takes $7.3M. We have invested $2.3M so far.
$2.3M invested
Goal $7.3M
The current goal is to reach an investigational new drug application (IND). Every gift has the potential to yield significantly increased funding through grants and investors.
โI canโt overstate the importance of advocacy. We have to fight to get attention, to get money to fund the studies, to find more and more families that feel theyโre living alone, so they can come together and be part of a community.โ
Caroline Hastings, MD UCSF
GM1 Gangliosidosis Community Stories
Our Trusted Partners & Awards
Take action and join us
Participate and take action
Our work depends on families sharing their data, on volunteers, and on donors. Find your lane below.
Affected by GM1
Start here
GM1 Families: Start Here
What to do in the first weeks after a diagnosis, in one place.
Join the GM1 Matrix
Your familyโs data is what makes a treatment provable. This is the single most valuable thing a family can do.
GM1 Clinical Trials Guide
What is open, who qualifies, and how to ask.
Annual GM1 Community Conference
Presentations on research, clinical trials, and caregiving, plus the families who understand.
Caregiver Support Group
A private group for GM1 parents and caregivers.
About GM1
What GM1 is, how it progresses, and what is being tried against it.
Share your story
Your familyโs story is what moves researchers, funders and lawmakers.
Want to help
What helps most
Give monthly
Predictable funding is what lets us commit to research that takes years.
Start a fundraiser
Turn a birthday, a race, or any special occasion into research funding. Reach the people we cannot reach ourselves, and we send you the kit.
Advocate with us
Newborn screening, FDA engagement, and awareness work. Much of our progress is volunteer driven.
Shop the GM1 store
Wear it, and start the conversation for us.
Join Friends of Cure GM1
The Facebook group for supporters, not just families. Where fundraisers, awareness pushes and volunteer asks get shared first.
Share a quote
A few words from you, used in our awareness and advocacy work.
Get the research newsletter
Plain language updates on GM1 science, a few times a year.
Our Blog
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GM1 Gangliosidosis vs. GM2 Gangliosidosis (Tay-Sachs / Sandhoff): Understanding the Differences
From First Clinical Descriptions to Modern Therapeutics, a plain-language guide for families and caregivers Why…
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How to Help Fund Research and Support the Cure GM1 Foundation
GM1 gangliosidosis is a rare, inherited disease that robs children of their abilities, and their…
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GM1 Gangliosidosis: A History
GM1 Gangliosidosis: A History From First Clinical Descriptions to Modern Therapeutics Last updated August 2026…
GM1 and rare disease communityโ
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 Administration, FDA.
Jenny Bragg,
GM1 parent
โIf 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.โ
Kylie Harrison,
GM1 Parent
โPeople 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
โCure 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.โโ
Emil Kakkis, Advisor
โI 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.โโ
Why Cure GM1 exists
GM1 was identified in 1959. For fifty years, almost nothing happened.
GM1 gangliosidosis was first identified in 1959, 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 was founded to end that. Here is what eleven years of focused advocacy and funding have changed.