Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
GET INVOLVED
Happy Easter and Happy Spring!
GM1 Day is May 23, and you can help us make this year our most powerful yet! Our community may be small, but together, we can make a mighty impact for those living with GM1. See below for information on requesting a GM1 Day proclamation in your state or city, as well as new GM1 Day swag, social media downloads and more. Got other ideas? Message us! We’d love to hear your plan for bringing awareness to GM1!
This year especially, we have so much to be grateful for, thanks to this community’s continued support and advocacy efforts. Below you’ll find updates on:
- A new fetal GM1 trial involving gene therapy
- A new (paid!) clinical outcomes assessment study for Type 2 families funded by FDA
- Opportunities to contribute to the Cure GM1 biorepository through our partner CombinedBrain’s collection sites around the country.
Quick reminder: Cure GM1 is back on X.com (@curegm1), so please consider following us on X and on all other social platforms to ensure our voice continues to be heard.
And, don’t forget: This year’s virtual International Cure GM1 Community Conference registration is open! Sign up now to secure your free access to this year’s event.
Together, we are making progress. We are so grateful for your commitment to making these advancements possible.
Community Engagement
Reece’s Story
“Reece, we are told, is a miracle for still being with us at age 34. But this has not been easy.“
In this issue, the mother of Reece shares his story of living with GM1 Type 2 since age 3.
Early Registration for the 2026 conference is open!
Registration for this year’s virtual International Cure GM1 Community Conference is now open and FREE for GM1 Families. Don’t miss this opportunity to spend the day learning more about advancements and research being done in the GM1 space.
Click here to check out the conference website.
Mail info@curegm1.org for a sponsorship package.
New Cure GM1 Blog is Up and Running
We share a lot of information, and we want you to be able to find and reference it as easily as possible. That’s why we’ve launched a new blog on the Cure GM1 website. The blog is already packed with useful information to better understand GM1 and to navigate life with GM1. This month, we added recaps of past conference speakers who made a significant impact on our attendees. This blog will continue to be updated with new stories monthly, including:
How to Handle More Than You Can Handle
Canine Companions and Their Impact on Special Needs Families
Creating a Special Needs Action Roadmap.
Welcome to Our New Cure GM1 Board Members
Cure GM1 is proud to welcome Josh Henderson and Varuna Gopalan to the Cure GM1 Board of Directors. We also express our gratitude to board member Gouri Yogalingam, Ph.D., who will now be transitioning to a scientific advisory role.
Learn more about Josh, Varuna and Gouri here.
Don’t Miss the Next GM1 Community Chat: Thursday, April 23, 2 p.m. PT / 5 p.m. ET
This is your chance to connect with other GM1 families, ask questions, and find honest feedback and support. Registration is required to ensure the safety of our members.
Advocacy
Request a GM1 Day Proclamation in Your State!
Every year, we work to bring greater awareness to our mission via GM1 Day, May 23. One of the most integral parts of GM1 Day is the act of having governors and mayors around the country make an official declaration of GM1 in their state or town. We currently have proclamations in the works for California, Florida, Idaho, Illinois, Texas, Virginia, New York, Georgia, Colorado, Wisconsin, Iowa, Georgia and Puerto Rico. Do you or your family members and friends live in a state not listed here? Let us know! We can help you fill out the short online form to bring GM1 Day to your area. Email jess@curegm1.org for information.
Check Out This Year’s GM1 Day Web Page
There are tons of easy ways to support Cure GM1 this GM1 Day. Check out our official GM1 Day web page for official GM1 Day merch, social media downloads, fundraising ideas and more.
Moving Mountains for GM1? Apply for the Sanofi TORCH Award!
Each year, the Sanofi TORCH Awards honor those who have made a difference in the rare disease and rare blood disorders communities. The Cure GM1 community has been recognized multiple times for its important work raising awareness of GM1. Past winners include Board member and parent David Law (2024); board member and parent Kylie Harrison and husband Kyle (2021); parents Ryan and Jenny Bragg (2018); parent Marilee Leishman; and Cure GM1 Co-Founder Christine Waggoner (2017).
Nominations are due by April 10.
Click here to nominate yourself or someone else now!
Calling All Runners!
An ultra-running opportunity to support our ultra-rare GM1 community! The Javelina Jundred has charity bib openings for both their 100-mile and 100-km races! This is an opportunity to participate an in iconic ultra marathon while raising funds for the Cure GM1 Foundation! The race takes place Oct. 31-Nov. 1 in Fort Mcdowell, Arizona. Click below for more information! Runners must commit to raising $4,000 for the 100m and $3,000 for the 100k.
New Share-able ERT Videos Available on YouTube
Check out the Cure GM1 Foundation’s new “shorts” regarding Enzyme Replacement Therapy, now available on our YouTube Channel. These videos will help educate and raise awareness of GM1 and the promise of ERT.
research & biotech news
New Phase 1 Trial for Fetal GM1 Gene Therapy Just Announced
A Phase I study of prenatal intravenous gene transfer with an AAV9 vector has been announced for both Type 1 and Type 2 GM1. If parents are identified as genetic carriers of the Type 1 or Type 2 gene, a GM1 diagnosis will be determined in utero to determine eligibility for the study.
GM1 Type 2 Families Invited for Paid Study on Caregiving
The Cure GM1 Foundation is supporting recruitment for a global research study conducted by Sprout Health Solutions on behalf of the Critical Path Institute. The study is funded by the FDA with a goal of learning more about your experience as a GM1 caregiver.
Participants must be primary caregivers of children living with GM1 Type 2a (late-infantile) or Type 2b (juvenile) or bereaved primary caregivers whose child with GM1 passed away within the past 12 months. If you are eligible, you may be invited to take part in paid interviews about your experience.
Or, email GM1-study@sprout-hs.com.
Share GM1 Data Via New Biorepository Partnership with CombinedBrain
Cure GM1 is teaming up with biorepository leader CombinedBrain to accelerate research of GM1. CombinedBrain helps advance rare disease research by collecting, storing, and distributing high-quality patient samples (blood, tissue) to researchers worldwide. The goal, ultimately, is to enable potential development of treatments for GM1.
See below for a list of collection locations. Please message us directly at info@curegm1.org if you need help getting involved.
FDA RISE Workshop Welcomes Cure GM1 Board Member as Panelist
Cure GM1 board member Vanessa Rangel Miller was a panelist for the March 30, 2026, FDA RISE workshop on data sharing in the rare disease community. The public workshop was a co-project of the Duke-Margolis Institute for Health Policy and the U.S. Food & Drug Administration. Topics of the discussion included informed consent, data sharing agreements, legal issues and data quality.
Fundraisers
Spring Has Sprung for Cure GM1!
As we head into GM1 Day May 23, spring is the perfect time to organize a fundraiser of your own for the Cure GM1 Foundation. Whether you organize a community run, barbecue, or social media fundraiser, your support will help us move even closer to making enzyme replacement therapy a reality. Message jess@curegm1.org for your personalized fundraising page.
And don’t forget: we still have tons of goods available on the Cure GM1 store, including Mother’s Day gear, chocolates and flowers. Shop now.
Get Involved
There are So Many Ways to Get Involved with Cure GM1!
Email jess@curegm1.org if you would like to participate in any of the following:
- Birthday wishes for your child
- Blog or share your GM1 family story
- Plan a fundraiser, event or find another creative way to help.
We appreciate everything you do for GM1! It takes all of us.
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