Cure GM1 Catalyst | August 2026
Hello! We have some incredible news for the GM1 community. In July, members of the Cure GM1 team met with representatives of the U.S. Food & Drug Administration (FDA) to discuss the future of Enzyme Replacement Therapy (ERT) as a treatment for those living with GM1 gangliosidosis.
Holding this meeting required us to submit extremely detailed information regarding the current status or our ERT project, as well as detailed plans to move it forward. This was a very significant milestone and will pave the way for the next steps in the process. (See below for a brief graphic overview of the FDA drug approval process.)
We know how tiring it can be to wait when our cause is so urgent. But we are fulfilling our commitment to advancing ERT as a treatment for GM1. We want you to know this meeting left us feeling supported and positive about this path.
We hope you will help us keep this positive momentum moving forward. Please see below for more ways to get involved and help us make history, from joining the new GM1 MATRIX to fundraising and sharing the news on social media. Drug development is incredibly costly, but together, we can make it happen. It’s critically important to push forward, no matter what the challenges.
Countless generations of GM1 children deserve a better life. I believe we can all play a part in making that future possible.
Thank you for being part of this community!
Christine
Community
Florence’s Story: An Update
“I think the biggest surprise regarding the progression of the disease is that it’s not linear or predictable. It always keeps you on your toes. It’s a real rollercoaster.”
-Florence’s Mom
Back in 2019, we shared the story of Florence, who was diagnosed with GM1 at age 2. Here, her mom shares how Florence is doing seven years later.
Click here to read Florence’s story now.
Join the GM1 Matrix
Cure GM1 Foundation has initiated data collection through the Matrix platform to collect natural history data and real-world evidence to support the development of possible treatments for GM1 gangliosidosis. It is imperative that our families share and participate in this data-sharing opportunity.
Upcoming efforts to fund and develop ERT rely heavily on patient data. We need all hands on deck!
Click here to register and share data.
Click here to schedule a 1:1 meeting to walk through the process.
New Blogs Just Published!
We are working hard to develop educational content about GM1 to make it easier to engage with family and friends about this ultra-rare disease. Recent posts include “How to Help Fund Research and Support the Cure GM1 Foundation” and “GM1 Gangliosidosis: A History.” Both pieces are incredibly informative and way ways to learn and spread awareness of GM1.
Click to view our whole blog library.
Cure GM1 Community Chat: Thursday, Aug. 13, 2 pM PDT/5 PM EDT
Please join us! This monthly gathering is a casual opportunity to connect with other families who understand the GM1 journey. You don’t have to move through this experience alone. We’re here for you!
New to the community? You can share as much or as little as you feel comfortable with and join calls as often you like! You can always turn off your camera if you feel more comfortable. These calls are all about getting what YOU need to feel less alone.
Every meeting is different, as topics are guided by the group’s needs. Registration is required to ensure participant privacy. This is a FREE event.
Click here to register now.
Advocacy
Register NOW for the 2026 international GM1 Community Conference
We can’t wait to see you Oct. 2 for this year’s conference. We know October seems far away, but it isn’t (at least not for our conference planning team!) We are already working hard to develop content and recruit speakers that will make a meaningful impact on our families. Reasons to attend:
- New breakout sessions will allow you to make personal connections with other families
- Only registered conference attendees will have access to the complete set of conference content following the event
- You’ll learn about the latest in GM1 research first-hand from biotech companies and have the chance to ask questions throughout the day
The conference is FREE of charge for GM1 families. Registering early takes just a moment but helps us tremendously.
Global Genes Symposium for RARE Drug Development Sept. 9-11
Global Genes, in partnership with Boston Children’s Hospital and the Termeer Institute, will be hosting the annual RARE Drug Development Symposium in Boston Sept. 9-11. The 2.5 day symposium is designed to equip advocates, industry leaders, and academic experts with the tools to navigate early-stage research.
Click here to register.
research & biotech news
Azafaros: Enrollment Complete for NAVIGATE Phase 3 Nizubaglustat Study
Azafaros says it has completed patient recruitment in its Phase 3 study of nizubaglustat for patients with GM1 (late-infantile and juvenile) and GM2 gangliosidosis. Now that enrollment is complete, the company will begin to generate clinical data to evaluate the potential of using nizubaglustat to alter disease progression in GM1. The Phase 3 study required a minimum of 75 patients across 25 clinical sites in 13 countries and will last 18 months. Some patients may receive a placebo. Those treated will be eligible to continue nizubaglustat following the study period.
Click here to learn more.
Study Shows Advanced Brain Age in Patients with GM1 Type 2
Results of a study released in July 2026 showed that predicted brain age in late-infantile and juvenile GM1 patients will reflect clinical disease progression in GM1. The study used machine learning and MRI scans to assess neuronal degeneration in both GM1 patients and neurotypical controls. While a neurotypical control group showed whole-brain aging at a rate of 0.83 per chronological year, those with juvenile GM1 showed deterioration of 1.57; those with late-infantile showed deterioration of 12.25. Moving forward, this sort of analysis could help in determining disease progression or evaluating potential treatment.
Click here to learn more.
Get Involved
Share Your Photos and Stories for the 2026 Conference!
Each year, we put together a video that reflects the heart of our community: our families living with GM1. Please click below to share a photo and quote to be part of this year’s event and other future Cure GM1 Campaigns! Thank you in advance for sharing!
Click here to upload now.
Fundraisers
Do It for GM1 Fundraiser Moved to September
To better serve the needs of our community, we are moving Do It for GM1 from August to September. You can still sign up now and start planning how you’d like to participate!
Click here to join our team.
Shop the Cure GM1 Store!
Don’t forget: community members have year-round access to the GM1 store, where we offer a wide range of shirts, stickers, flowers and chocolate that help fund GM1 research.
Click here to shop now.
Ways to Give – Your Support Matters
RaiseRight | Walmart Spark Good | Facebook Fundraisers | Donate |
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