Cure GM1 Catalyst | October 2026
COMMUNITY
ADVOCACY CORNER
GET INVOLVED
SHOP FOR GOOD
Thank you for an amazing September! We are feeling deeply grateful for our community and your inspiring support during the Do It for GM1 campaign, which raised an astounding $34,804.78! Every dollar is going to work right away toward our $1.4M commitment to enzyme replacement therapy this year.
After all these years of fundraising, I’m still deeply touched by every bit of support. Your kindness matters to this cause, and this foundation has grown and changed because of it. Thank you to every team that took part and to every donor. Your dedication all month was amazing to watch.
We carried that energy right into October, kicking off the month on October 2nd with our annual conference. It was incredibly inspiring to learn about the latest research updates, connect with families, and experience the power of storytelling together.
Now our efforts turn to our natural history study on the Matrix. The Matrix is a study platform for gathering meaningful data, and it is a collaboration with CombinedBrain.
Why does it matter so much? Because when trials arise, robust natural history data is what lets our children avoid placebo. Every treatment approach and every trial needs that data before it can proceed. Strong data paired with an engaged community is what draws real investment into GM1. Ultimately, data is also what drives treatment approvals.
October is also Pregnancy and Infant Loss Awareness Month. This month we honor and remember the infants and babies whose lives GM1 cut short. Let’s celebrate their memories and bring awareness to the tremendous losses GM1 causes.
As always, thank you for your support. Read on for the latest news, our year-end plans, and more.
Sincerely,
Community
Milo’s Story
“Milo was a warrior. In so little time he taught us so much. He taught us to treasure what we love most, to enjoy it, to care for it. He was a warrior until his last days. And he taught us that even through the biggest obstacles he always carried magic and peace.”
–Milo‘s Mom
Free companion to navigate GM1: Basion
Keeping track of a child’s care with GM1 means a lot of paperwork: letters, lab results, scans, forms, and notes from appointments with many different specialists.
Through our partnership with Basion, GM1 families can now use a free tool that helps carry some of that load.
Basion organizes your child’s records into one timeline. When you’re wondering what a result means, you can ask, and Basion answers with cited sources. It also prepares summaries before appointments and lets you know about trials and support groups near you. Basion is HIPAA compliant.
Basion is free for GM1 families through Cure GM1. Sign up for Basion.
Survey on Prenatal Gene Therapy
A team led by Dr. Tippi MacKenzie at the UCSF Center for Maternal-Fetal Precision Medicine and the Center for Genome Surgery is planning a new clinical trial of in utero AAV9 gene therapy for GM1 gangliosidosis. They want to hear from GM1 caregivers as they plan.
Please share your opinion about prenatal gene therapy by taking the UCSF survey or scanning the QR code. You do not have to be a pregnant caregiver to take it. All caregivers are invited to participate.
New on the Blog
GLB1, GM1, Morquio B: do I have both diseases? A frequently asked question with an explanation that should be clarified to families.
Cure GM1 Community Chat: Thursday, Oct. 22, 2 p.m. PDT / 5 p.m. EDT
Please join us! This monthly gathering is a casual opportunity to connect for guidance and getting to know each other. 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.
Advocacy Corner
Conference Highlights
Nearly 100 people from 20 countries registered for this year’s conference, including about 70 members of GM1 families. Families registered from 18 of those countries. The US had the most registrants (58), followed by Italy (7), the UK (4) and Brazil (4). Researchers, clinicians, industry and advocacy partners, speakers and the CureGM1 team made up the rest. A total of 25 speakers and moderators participated. We created our own custom translation software this year which included both audio and captions.
- Recordings will be provided to everyone who registered. Select highlights will also be shared on YouTube or on our blog.
- If you attended, please fill out the conference survey.
- Thank you to our sponsors, speakers, board members and volunteers for a fantastic event!
First AAV Gene Therapy Approved for Sanfilippo Type A, a Lysosomal Disease Similar to GM1
On September 17, 2026, the FDA approved FAYUVI (UX111), a single-dose intravenous AAV9 gene therapy from Ultragenyx, for children with Sanfilippo syndrome Type A (MPS IIIA) whose neurodevelopmental function is still preserved. It is the first FDA-approved treatment for Sanfilippo Type A.
This video of a young girl named Tilly treated as a baby with FAYUVI shows the amazing potential when children receive gene therapy before irreversible damage has occurred.
GM1 gangliosidosis still needs our advocacy. No treatment for GM1 has yet advanced to approval. Let’s keep advocating and fighting!
Rare Disease Week Travel Reimbursement
The EveryLife Foundation has rebranded as the Rare Foundation. Its signature event, Rare Disease Week, takes place March 2 to 4, 2027, in Washington, DC. Travel reimbursement is available to help US advocates get there. Applications close Friday, November 6. Apply for Rare Disease Week travel reimbursement. Event registration opens January 6, 2027.
WORLDSymposium
Congratulations to Dr. Cynthia Tifft, on receiving the 2027 Roscoe O. Brady Award! We are grateful for your dedication and hard work.
Get Involved
Share Your Holiday Wish
This holiday season, we are building a series around GM1 families sharing, in their own words, what they wish others understood about life with GM1 and what they hope research will make possible. Your photos and quotes will be part of our social media and year-end communications.
What is your wish for your child or for the GM1 community this holiday season?
Share your wish and photos by November 30.
Fall and Halloween Fun
Follow our social media this month as we bring awareness to how nonverbal and disabled children navigate Halloween. And send us your Halloween pictures!
Let’s Finish Strong
Save the date! Giving Tuesday 2026 will take place on December 1st, falling on the Tuesday immediately following Thanksgiving.
More than one-third of the average nonprofit’s annual revenue comes in the final quarter of the year.
Please make a plan to join us by helping support our year-end campaign and Giving Tuesday. Mail info@curegm1.org to join the team.
Shop For Good
Special Halloween designs are now available in the GM1 store.
Fall bulbs are now available through Oct. 15.
Treat yourself or a friend year-round and benefit Cure GM1.
Ways to Give – Your Support Matters
RaiseRight | Walmart Spark Good | Facebook Fundraisers | Donate
Your gift is tax-deductible to the fullest extent allowed by law. We are a 501(c)(3) organization with minimal overhead—maximum impact goes directly to research. Tax ID: 47-3154419
Visit our Take Action page for more ways to support our community.