Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
IN MEMORIAM
It’s a brand new year! Every single passing moment, we could be closer to truly meaningful change for all those suffering from GM1. That’s why we’re putting Hope in Motion in 2026. Stay tuned for more updates on ways to get involved as the year moves on.
In 2026, we’re doubling down on efforts to advance enzyme replacement therapy (ERT) as a promising potential treatment for GM1. In fact, we’re committing significant spending (~$1.4 million!) toward ERT this year alone which is the largest investment in a single project that we have ever made. This will be paid throughout the year as the project progresses.
This is an all-hands-on-deck effort! We cannot make ERT a reality without your help. In the coming year, we’ll be announcing new and exciting opportunities to play a role in this potentially life-altering treatment program. This includes things like:
- Sponsorship opportunities
- Planned giving (estate planning) opportunities
- Monthly and yearly pledge plans
- Personalized community fundraising pages
Why the rush? First, our children’s lives depend on it. The faster we develop this treatment, the sooner they could potentially benefit. Second, the costs of developing ERT to complete an investigational new drug application (IND) will dramatically increase over time. The more we can “de-risk” the program by helping demonstrate its viability, the better our chances for long-term investment through grants and/or partnership.
We look forward to sharing more news on ERT as the year progresses! In the meantime, please reach out to info@curegm1.org to get involved in this incredibly promising effort! Or, click here to donate now.
Our 2026 Impact Report is now available!
Click below to view it now.
Community Engagement
Illinois GM1 Family Receives Pup as Part of Make-a-Wish Experience
Ezra, a 2-year-old living with GM1, recently received a Make-a-Wish experience related to his GM1 gangliosidosis diagnosis. Ezra’s family chose a pup named Norman as Ezra’s Make-a-Wish gift. Mom Ashley Thornton recently shared Ezra’s story with the local news. Click to read their story or click here to watch a video about the experience.
Join Our Board of Directors!
Cure GM1 is actively seeking dedicated individuals to join our Board of Directors in the new year. If you’re looking for a meaningful way to give back and help guide a mission-driven organization making real change, we’d love to hear from you. Learn more and apply by mailing info@curegm1.org for details.
Don’t Miss the January Community Chat
Our Community Chat for GM1 parents is a safe space to share your experiences and connect with other families living with GM1. This month’s Community Chat will take place Thursday, January 15, at 4 p.m. PST/7 p.m. EST. Click here to register.
Advocacy
We Must Resume Our Advocacy in 2026 to Support the Rare Pediatric Disease Priority Review Voucher (PRV) Program
The U.S. Senate attempted to pass the Give Kids a Chance Act (S. 932) including re-authorization of the Rare Pediatric Disease Priority Review Voucher (PRV) Program, but the vote did not succeed. The act required a unanimous vote in the Senate in order to fast track approval. While extremely disappointing, the momentum from our rare disease advocacy community remains strong, and important progress has been made.
When the Senate returns in January, advocacy will resume with even greater strength, and the work will not start from scratch. We urge the Senate to act swiftly to pass this legislation and protect life-saving innovation.
Learn how to engage your Senators and access advocacy resources here: https://everylifefoundation.org/prv/
We Need Your Stories for Rare Disease Day!
Every year, Rare Disease Day helps to elevate awareness of GM1 gangliosidosis on the global stage. Personal stories always make the greatest impact. If you feel comfortable sharing your family’s experience living with a GM1 diagnosis, please email jess@curegm1.org.
You can send photos, quotes, a brief history of the diagnosis process or anything else you feel the public should know about GM1. Photos and videos of hospital stays, doctor visits, new/worsening health challenges, before/after progression are especially impactful. These stories will be shared online and in potential fundraising campaigns. Thank you in advance for helping to get the word out.
No Patient Left Behind Signs FDA Letter
No Patient Left Behind (NPLB), a network of healthcare investors, researchers, professionals and patients, has drafted a letter of concern to the U.S. Food and Drug Administration Commissioner Martin Makary. The letter demands a return to evidence-based policy, among other concerns. Read NPLB’s letter here or click here to sign the letter.
Check Out Our 2026 Annual Calendar
Don’t forget to check our new 2026 annual calendar and plan ahead our major events and dates, including opportunities to advocate on behalf of those living with GM1. Click here to download a printable version of the 2026 calendar now.
Free Registration for GM1 Families at WORLDSymposium 2026
If you will be in the San Diego, California, area Feb. 2 to 6, consider attending this year’s WORLDSymposium on lysosomal diseases. This year’s preliminary program includes numerous sessions regarding research on GM1, including an Azafaros talk on the phase 3 18-month placebo-controlled study focusing on GM1, GM2 (Feb. 3); therapeutic applications for glycan biomarker H3N2b in GM1 (Feb. 4); and the NIH AAV9 gene therapy for GM1 Type 2 (Feb. 6). In addition, Cure GM1’s Christine Waggoner will be presenting on the Cure GM1 Census and Patient Registry.
Please note: Presentations shared at WORLD Symposium are aimed at members of the scientific community. WORLD also offers complimentary registration for GM1 families.
To request your complimentary registration now, click here.
research & biotech news
Lentiviral Hematopoietic Stem Cell Gene Therapy Improves Outcomes of GM1 Gangliosidosis in Mice
Researchers have recently completed a study that illustrates the use of Hematopoietic Stem Cell (HSC) gene therapy can improve the outcomes of mouse models with GM1. HSC gene therapy has already been proven effective in treating numerous lysosomal storage diseases, and in the recent GM1 study, it “broadly restored enzymatic activity and reduced the lysoosomal storage in the brain.” This, in turn, improved neuromuscular symptoms and overall animal survival.
To read the full article, which will be featured in Molecular Therapy click here.
This particular approach was recently highlighted in the news due to success in MPS II / Hunter Syndrome. One of the primary investigators involved in the creation of this therapy is an advisor to Cure GM1. Congratulations to Dr. Simon Jones and team! Click here to read the article.
Azafaros Adds Biotech Leader to Board
Azafaros, which is leading a Phase 3 clinical trial regarding the use of nizubaglustat for GM1, recently announced the addition of biotech entrepreneur Dr. Thierry Abribat to its board. To learn more, click here.
Fundraisers
Create a Personalized Fundraising Page for Cure GM1
This year, we’re making it easier than ever to fund raise on behalf of the Cure GM1 Foundation. Simply reach out to jess@curegm1.org, and we’ll create a fundraising page personalized for your family. Just send your child’s name, photos and/or videos featuring your child, a bit of background you feel comfortable sharing about your child’s experience with GM1, and a fundraising goal if you have one. We’ll take care of the rest. Request a fundraising page now.
Order now for Rare Disease Day!
Help raise awareness of GM1 gangliosidosis this Rare Disease Day with custom GM1 t-shirts, signs, bumper stickers and other swag! This year, we highlight the phoenix. Just like the rare and magical phoenix, every child living with GM1 is a symbol of resilience, strength and hope.
Order now through Jan. 14, and you’ll receive your shirt by Feb. 2. Click here to order now.
And: Other New Merch Available
Help spread awareness of GM1 gangliosidosis with one of our many merch options, including this new sticker. Find it and other items now at the GM1 Store.
Spring Flower Power Fundraiser is Open!
When you buy your spring flowers at Flower Power, you help support Cure GM1 while you shop. This spring’s assortment includes a wide range of beautiful and colorful flowers, from lilies and dahlias to orchids, daisies and geraniums. Click here to shop now.
In Memoriam
We are extremely saddened to share that in December, three young children in our community passed away in the span of 4 days. We extend our deepest condolences to the families of Dawson, Kassidy and Amelia.
Annual Candlelight Memorial
We hope you were able to join us Dec. 14 as part of the Compassionate Friends Candle Lighting Memorial in remembering those beautiful children lost to GM1 gangliosidosis in 2025. In case you missed it, the 2025 video, as well as other memorials from past years, can be found on our website. Click here to view it now.
Please know this video accounts for just a fraction of those who have died from GM1 this year. If you know of anyone we missed, please send us a note at jess@curegm1.org.
Ways to Give – Your Support Matters
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Set up a Recurring Donation
Visit our Take Action page for more ways to support our community.
Cure GM1 Foundation | PO Box 6890 | Albany, CA 94706 US