Cure GM1 Catalyst | November 2026
COMMUNITY
ADVOCACY CORNER
GET INVOLVED
SHOP FOR GOOD
This year, Iris turned 18. She is still waiting for an approved treatment, and so are so many of the children and adults whose faces fill our community. But Iโm writing to you this November with something I havenโt always been able to offer: real, concrete momentum.
In July, our team sat across the table from the U.S. FDA for a formal INTERACT meeting about our enzyme replacement therapy (ERT) program. For a rare mom, thatโs a moment you hold onto. ERT already helps people with other lysosomal diseases, but it has never reached a GM1 clinical trial. Weโre working to change that, and the FDA is now in an ongoing conversation with us about how to get there.
The next step is an IND-enabling study, the first step of the safety work required before a trial can begin. We need to raise $600,000 by January to fund it.
Hereโs the part that gives me so much hope. From November 1 to December 15, our Board of Directors will match every gift, dollar for dollar, up to $25,000. Our board is all in. Iโm asking you to match their commitment and go as far as possible to help fund the next step in our ERT work.
Youโve already shown up in such big ways this year. Do It for GM1 raised $34,804.78 for ERT. Nearly 100 people from 20 countries joined our conference in October. Eight states and three cities proclaimed GM1 Awareness Day. And there have been so many other daily ways you have shown up for each other and our cause.
We also honor every child weโve loved and lost by keeping this work moving. Your gift today does exactly that. We invite you to submit quotes and photos for the Compassionate Friends global candle lighting in December. Read more below.
Sincerely,
Community
Remembering Theodore
Theodore would have turned five this year. His family has shared a beautiful remembrance video in his honor. We carry him, and all of them, with us as we keep fighting.
National Family Caregivers Month
November is National Family Caregivers Month. The Courageous Parents Network has a free resource bundle to support families caring for children with serious illness. No effort in caring for your child is too small, and you donโt have to navigate this alone.
New on the blog
Read the full recap of our October conference, including research updates, speaker highlights, and whatโs coming next.
Cure GM1 Community Chat: Thursday, Nov. 19, 3P.m. PST/6P.m. EST
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.
Thank You, Rosie’s Community!
A heartfelt thank you to Rosieโs family and friends, who held a bake sale to support Cure GM1. Your generosity and creativity mean so much to all of us. Thank you for showing up for this community!
Advocacy Corner
Remembering Dr. Jagdeep Singh Walia
We are deeply saddened to learn of the sudden passing of Dr. Jagdeep Singh Walia on October 20, 2026. Dr. Walia was a Canadian medical geneticist and researcher at Queenโs University whose work contributed to the development of treatments for GM1 and GM2 gangliosidoses. Of particular importance to our GM1 community was his collaboration with Azafaros on nizubaglustat, an investigational oral drug in Phase 3 clinical development for GM1 and GM2 gangliosidoses. His laboratory contributed to promising preclinical research supporting this treatment approach.
For families affected by GM1 gangliosidosis, the loss of a scientist working to advance potential treatments is particularly meaningful. We extend our heartfelt condolences to Dr. Walia’s family, colleagues, and everyone whose lives he touched.
FDA launches rare disease drug development framework
The FDA has introduced a new framework designed to improve and streamline the drug development process for rare diseases. The framework outlines how the agency will engage with sponsors of rare disease therapies earlier and more consistently, with the goal of reducing barriers to approval.
This is meaningful news for our community. Our INTERACT meeting this past July is exactly the kind of early engagement this framework is designed to support. GM1 gangliosidosis still needs our advocacy. No treatment for GM1 has yet advanced to approval. Letโs keep advocating and fighting!
Azafaros PRONTO natural history study published
A significant new paper has been published: โA prospective, longitudinal, natural history study of neurological disease trajectory in children with late-infantile or juvenile-onset GM1 gangliosidosis or GM2 gangliosidoses: the PRONTO study.โ Natural history data like this is foundation for advancing treatments. When trials arise, this kind of robust data is what gives our children the chance to receive the treatment rather than a placebo.
Shionogi acquires Intrabio
Shionogi has agreed to acquire Intrabio, a commercial rare disease biotech, for up to $2 billion. Intrabio has programs in lysosomal storage diseases. We will continue to monitor how this acquisition affects the broader rare disease landscape.
Read more about the Shionogi-Intrabio acquisition.
Get Involved
Double Your Impact This Giving Tuesday
Mark your calendars for Giving Tuesday and help us make this the most impactful one yet for our GM1 families.
To kick off this season of giving, our Board of Directors is matching all donations dollar for dollar, up to $25,000, from now through December 15th.
We need to raise $600,000 by January to fund the IND-enabling study that is the next step toward an ERT clinical trial for GM1. More than one-third of the average nonprofitโs annual revenue comes in the final quarter of the year. Your gift now goes twice as far. Please make a plan to give before Dec. 15.
Compassionate Friends Global Candle Lighting
The Compassionate Friends worldwide candle lighting will take place on December 13, 2026 at 7:00pm local time. Families around the world light candles together in memory of children who left too soon. We invite bereaved GM1 families to take part.
Learn more about the candle lighting.
Submit Photos and Quotes for the Annual Memorial Video
We honor our children by keeping their memory present in this community. If you would like your child included in our annual memorial video, please share a photo and a quote or message. To see how our GM1 loved ones have been remembered in past years, watch our previous In Memoriam videos.
Submit your photo and quote here.
Share Your Holiday Wish
This season, weโre amplifying the voices of GM1 families. Tell us, in your own words, what you wish people understood about life with GM1 and what you hope research will make possible. Your words and your childโs photo will be part of our social media and year-end communications.
Share your voice by November 30.
Shop For Good
New holiday designs are now available in the GM1 store.
Holiday bulbs are now available through Dec. 15.
Gift sweet moments this season while supporting Cure GM1.
Cards and Prints
Send beautiful holiday cards this season through Minted! Use code FUNDRAISECGM1 for 20% off your order, and Minted gives 10% back to Cure GM1. A gift that gives twice.
Give the Gift of Gift Cards with RaiseRight
โTis the season for gift cards, and RaiseRight makes every purchase count for GM1. Enroll today and a percentage of every gift card you buy comes back to Cure GM1 at no extra cost to you.
Learn more at curegm1.org/raiseright or enroll here.
Ways to Give โ 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 overheadโmaximum impact goes directly to research. Tax ID: 47-3154419
Visit our Take Action page for more ways to support our community.