GM1 gangliosidosis is a rare, inherited disease that robs children of their abilities, and their lives. Caused by a mutation in the GLB1 gene, it eliminates an enzyme that cells need to clear a toxic waste product called GM1 ganglioside. Without that enzyme, the substance builds up in the brain and organs, causing progressive neurological damage. There is no approved treatment. No cure.
The Cure GM1 Foundation exists to change that. Founded by families affected by this disease, Cure GM1 funds research, builds the patient community, and assembles the scientific and regulatory infrastructure needed to bring treatments to clinical trials. But none of that work happens without support from people like you.
Here’s how you can make a real difference.
Fund the Research Directly: Donate
The most impactful thing you can do is donate. Nearly all of the funds Cure GM1 raises go directly into research. That includes pre-clinical studies, grants to scientists worldwide, and the infrastructure that supports drug development. Money is what drives research, and what limits it.
If you’d prefer to give in a way that doesn’t involve a direct payment, there are options:
- Gifts of stock, often a tax-advantageous way to give
- Used vehicles. Donate a car (it doesn’t have to run)
- Corporate matching. Many employers match charitable donations, and some even triple them. Check with your HR department.
- Crypto. Cure GM1 accepts cryptocurrency through Every.org
- PayPal Giving Fund. Give through PayPal’s charitable platform
- Monthly recurring gifts. Sustained, predictable funding lets the team plan further ahead
Cure GM1 is a 501(c)(3) nonprofit (EIN: 47-3154419) and holds Candid’s Gold Seal of Transparency.
Have questions about giving options or want to discuss a specific type of donation? Get in touch and the team will be happy to help.
Support the Enzyme Replacement Therapy Project
Right now, Cure GM1 is advancing one of the most promising new approaches in the treatment landscape: enzyme replacement therapy (ERT) for GM1.
Current treatments being investigated, including AAV gene therapy and small molecule drugs, each have meaningful limitations. Gene therapy requires intervention before significant neurological damage occurs, but most children are diagnosed too late. Small molecule drugs need some residual enzyme activity to work, which means they can’t help children with the most severe form of GM1.
ERT works differently. It delivers the missing enzyme directly, without relying on any existing enzyme activity. That means ERT has the potential to help the majority of GM1 patients, including those who cannot benefit from other approaches. Precedents in other lysosomal storage diseases, like Aldurazyme (MPS I) and Brineura (CLN2 Batten disease), show that this approach can transform outcomes in diseases that are biologically similar to GM1.
Cure GM1 has assembled a team with decades of prior ERT experience, including veterans of BioMarin and other rare disease biotechs. The current goal is to raise $5M more to advance toward an IND filing, the regulatory milestone that opens the door to clinical trials in humans.
Your donation to the ERT project funds real, specific science: laboratory work, manufacturing development, and regulatory preparation. Every dollar brings the first GM1 enzyme replacement therapy one step closer to children who cannot wait.
Participate in the GM1 Matrix: Give Your Data
Research cannot happen without patients. With a disease as rare as GM1, every family who participates in data collection makes a meaningful difference. That’s why Cure GM1 launched the GM1 Matrix, a comprehensive natural history research study conducted in partnership with COMBINEDBrain.
The GM1 Matrix collects information from patients and families worldwide: subtypes, diagnostic journeys, symptoms, quality of life impacts, and medical records. This data is the roadmap that scientists need to understand the disease, design clinical trials, and prove that treatments are working.
Who can participate? Parents, legal guardians, or affected adults who can complete an online survey. All published data is anonymized. Participants own their data.
The more families who join, the faster research can move. Register now at nhs-study.curegm1.org.
Host a Fundraiser
When you fundraise for Cure GM1, you do something the foundation can’t do on its own: you bring the cause to your network. People trust you. They are far more likely to give when someone they know asks than when an unknown organization does.
There’s no shortage of ways to fundraise. Simple options include birthday fundraisers on Facebook, holiday campaigns, or online fundraisers through platforms like GoFundMe, JustGiving, or eBay for Charity. More ambitious ideas, like golf outings, fun runs, trivia nights, bake sales, or auctions, can bring your whole community together.
Cure GM1’s Fundraising Manual walks you through everything: how to plan your event, tell your story compellingly, build a publicity timeline, and maximize donations. The foundation can also help with logo use, an authorization letter, personalized fundraising pages on curegm1.org, and donor tax receipts.
A few tips from the manual that make a real difference:
- Tell an honest, vulnerable story. Facts inform, but stories move people to act.
- Set an ambitious goal. People who aim higher raise more.
- Post consistently on social media in the weeks leading up to your event.
- Mention corporate matching. Some donors can double or triple their gift this way.
Support While You Shop
Several retailers make it easy to direct a portion of your everyday purchases to Cure GM1:
- RaiseRight. Earn money for Cure GM1 when you buy gift cards
- Walmart Spark Good. Round up your Walmart purchases
- eBay for Charity. Designate Cure GM1 when you buy or sell
- GM1 Store. Shop for GM1 awareness gear and merchandise at curegm1.org/gm1-store
You can also support through Cure GM1’s vendor partnerships: See’s Candies and Flower Power Fundraising both have programs where proceeds benefit the foundation.
Spread Awareness on Social Media
Awareness creates the conditions for donations. When more people know about GM1, more people give, and more biotech companies take notice. Follow Cure GM1 on Facebook, Instagram, TikTok, LinkedIn, and YouTube. Share posts. Tag #curegm1. Post photos wearing GM1 gear. These small acts add up.
Participate in Research Directly
If you or a family member has GM1, there are meaningful ways to contribute beyond donations:
- Join a clinical trial. The more participants, the faster treatments become available.
- Participate in natural history studies. Your data helps researchers understand the disease’s progression and compare it against future treatments.
- Join the GM1 Biobank. Tissue and biological samples are critical to drug development.
- Sign a media waiver and share your story. Photos and personal stories humanize this disease in a way that data alone cannot. Submit your story or share a quote at curegm1.org.
Stay Connected
Subscribe to the Cure GM1 Catalyst newsletter to stay current on research progress, awareness campaigns, and community news. Attend the annual GM1 Community Conference, where patients, families, clinicians, and researchers come together to share what’s working and what’s next.
Join Our Private Facebook Groups
- Connect with other families by joining our private Facebook Group for parents and caregivers of those with GM1 and adults with Type 3 adult-onset GM1.
- Supporting someone with GM1? Join our Friends of GM1 Group specifically for extended family members and friends.
Join Events and Awareness Campaigns
Cure GM1 runs a year-round calendar of events that connect patients, families, clinicians, and supporters. Showing up is one of the most visible things you can do for this community.
- Annual GM1 Community Conference. The conference brings together families, researchers, clinicians, and advocates for a day of science updates, community, and hope. Register at curegm1.org/conference.
- GM1 Awareness Day. Held every April, GM1 Awareness Day is a coordinated campaign to raise awareness across social media with GM1 stories, facts, and faces. Join at curegm1.org/gm1day.
- Do It for GM1. A community fundraising and awareness challenge. Pledge to do something, share it, and raise money for research. Learn more at curegm1.org/do-it-for-gm1.
- Monthly Community Chats. Cure GM1 hosts regular online gatherings where families can connect, ask questions, and hear the latest updates. Check curegm1.org for the current schedule.
- Rare Disease Day. Observed on the last day of February each year, Rare Disease Day is a global awareness campaign. Cure GM1 participates every year. Get involved at curegm1.org/rare-disease-day.
Every Action Matters
There is no cure for GM1 today. But there is a clear, scientifically grounded path toward one, and what stands between the research and clinical trials is funding, data, and community. Every donation, every fundraiser, every new family who joins the GM1 Matrix, every post shared on social media moves the needle.
Children with GM1 cannot wait. The science is ready. The team is in place. What comes next depends on all of us.
Take action today at curegm1.org/take-action
Frequently Asked Questions
GM1 gangliosidosis is a rare, inherited lysosomal storage disease caused by a mutation in the GLB1 gene. Without the enzyme this gene produces, a toxic substance called GM1 ganglioside builds up in the brain and organs, causing progressive neurological damage. There is currently no approved treatment or cure.
You can give directly at curegm1.org/donate. Cure GM1 accepts credit cards, PayPal, cryptocurrency, stock gifts, vehicle donations, and recurring monthly giving. Many employers also offer matching gift programs that can double or triple your contribution.
Yes. Cure GM1 Foundation is a 501(c)(3) nonprofit (EIN: 47-3154419) and holds Candid’s Gold Seal of Transparency. Donations are tax-deductible to the extent permitted by law.
The GM1 Matrix is a natural history research study run by Cure GM1 in partnership with COMBINEDBrain. It collects information from GM1 patients and families worldwide, including subtype, symptoms, and quality-of-life data. This data helps scientists understand the disease and design clinical trials. Families can register at nhs-study.curegm1.org.
ERT delivers the missing GLB1 enzyme directly into the body without relying on any existing enzyme activity, giving it the potential to help patients at all disease stages. Cure GM1’s team is working toward an IND filing, the regulatory step that opens the door to human clinical trials. Your donation directly funds this work.
Download the Cure GM1 Fundraising Manual at curegm1.org for step-by-step guidance. Options include birthday fundraisers on Facebook, fun runs, auctions, and bake sales. Cure GM1 can provide a logo, authorization letter, and personalized fundraising page.