Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS
A note from Christine
Dear GM1 Community,
This month, our hearts are heavy as we said goodbye to another precious child, sweet Daxton, who passed away just before his fourth birthday. The heartbreaking reality is that most children with GM1 won’t see their fifth birthday—an unimaginable burden that no family should ever have to carry. We cannot and will not remain invisible in the face of this devastating disease.
This is exactly why our fight for enzyme replacement therapy is so critical, and why we need your help now more than ever. We’re launching our “Do It For GM1” campaign September 1st-30th — a new way for everyone to turn their passions into powerful fundraising for GM1 research. Whether you love running, baking, crafting, or anything else, you can transform what brings you joy into hope for our children. Will you make a plan to join us?
Our Annual Conference is just one month away, and it’s more than just an event—it’s where talks and content specific to GM1, planned specifically for our community are seen and heard. It’s where we connect with the researchers and experts who are working tirelessly to improve our children’s lives. From understanding your genetics report to learning about breakthrough treatments like ERT and gene therapy, this conference empowers families with knowledge and hope.
We need you. Register for our conference today and join the “Do It For GM1” campaign. Every voice matters, every story counts, and every action brings us closer to a world where GM1 children can be seen and celebrated for many more birthdays!
-Christine Waggoner
Community Engagement
Kiaan’s Story
Meet Kiaan and his inspiring journey through gene therapy for GM1. His mother’s heartfelt story reveals how his condition brought their family closer, uniting them in appreciation of every moment. Read here
In Loving Memory of Daxton
Our hearts are heavy as we honor precious Daxton, who peacefully passed away surrounded by his family’s love on June 18th, just a month before he would turn 4 years old. His mother’s beautiful words remind us of the profound impact these brave children have on all of us. Daxton will continue to inspire our mission to find treatments for GM1. We hold his family close in our thoughts and prayers during this difficult time. Read his story
Welcome, Ananda, to our community!
This sweet boy was born in May 2023 and was diagnosed with GM1 type 1 in April 2024. His diagnosis divided the family’s world into “before and after”. He was a joyful baby who loved to laugh, be held, and explore everything around him. Though GM1 has changed him, his parents love for him remains enduring, feeling his connection as they hold his hands. We’re honored to have you and your family as part of our GM1 community.
Introducing “Do It For GM1” – Our New Annual Fundraising Campaign
We’re excited to launch “Do It For GM1,” our new yearly fundraising campaign that builds on the success of Move For GM1 while expanding beyond fitness activities. Whether you prefer running, baking, crafting, gaming, or any other passion, you can now turn your favorite activity into a fundraiser for GM1 research. This inclusive approach means everyone can contribute in their own unique way while raising critical funds and awareness for our cause. Learn More & Start Your Own Fundraiser
2025 COMBINEDBrain Biorepository Roadshow
The CB Biorepository team is attending 9 patient conferences throughout 2025, making it easier for families to contribute vital research samples. If you’re interested in participating or know families who might be, reach out to their team. A staff member will contact participants to coordinate the process and answer questions. Learn More
ONE MONTH TO OUR ANNUAL CONFERENCE: DON’T FORGET TO REGISTER
Cure GM1 Annual Conference on August 7-8, 2025: Come and join us for a day of learning and connecting with other families and researchers. We hope to see you there! For those attending in person, we’ll host a welcome dinner on August 7. Check Out the Current Agenda
Keynote Speakers:
Amanda Griffith-Atkins is a licensed marriage and family therapist. Sixteen years ago, her newborn son was diagnosed with a rare genetic condition, and she discovered her life’s purpose: to help parents of children with disabilities find their stride. She’s the author of the book “How to Handle More Than You Can Handle: Caring for Yourself While Raising a Disabled Child“.
Hear the inspiring story of Kristin Gray. When her two daughters were diagnosed with Batten Disease, she found herself in the same situation that we, as GM1 families, go through: receiving a devastating diagnosis with no cure. Since then, she has founded the CureBatten foundation and has been pushing for a cure and is now initiating a new trial.
If you have any questions, reach out to us at info@curegm1.org
Have you checked our new family resources page? Let us know if you have any questions or feedback!
Cure GM1 Community Chat
You’re invited to our next caregiver chat amongst members of the global GM1 community! Join us at 9:00AM US Pacific Time on July 17th.
Advocacy
Cure GM1 Foundation at BIO International Convention
Christine and Gouri attended the BIO International Convention, one of the world’s largest biotech events gathering over 15,000 industry leaders. The event provided crucial networking opportunities to advance GM1 research collaborations and advocate for key priorities: renewing Priority Review Vouchers and securing increased funding for rare disease development. We’re grateful to our scientific advisor Dr. Gouri Yogalingam for joining us in advocating for GM1 families. These face-to-face connections are essential for moving GM1 research forward. Learn More
EveryLife Foundation’s Letter to FDA
Cure GM1 Signed EveryLife Foundation’s Letter advocating for enhanced FDA-patient advocacy engagement. While the FDA’s new CEO Listening Tour with pharmaceutical and biotech executives is a positive step, direct dialogue with patient organizations is equally crucial for rare disease therapy development. The letter requests town hall meetings to ensure patient voices are heard in regulatory policy. Read the Letter
Research America’s Letter to the NIH
Cure GM1 signed a community letter in support of the NIH. The letter garnered 500 organizations and 2,355 individual signatures – an extraordinary support! Read it here
Act for Ultrarare Congressional Event Replay Available
The “Act for Ultrarare: Shaping the Future of Drug Development” congressional briefing replay is accessible online. The event gathered advocates to discuss the barriers preventing treatment development for the smallest patient populations and highlighted critical challenges and potential policy solutions in drug development. Watch the Replay
Biotech News
Save the Date: Azafaros’ webinar on the upcoming Phase 3 of nizubaglustat clinical trial logistics
Save the Date: Jul 7, 2025 at 5 pm ET / 2 pm PT
Registration: NO registration needed. Watch Here
Dawn Blessing Presents Work on GM1 ERT
A very special thank you to our board member Dawn Blessing for presenting work on GM1 enzyme replacement therapy at the BioCentury Inc. Grand Rounds in Chicago. Learn More Here
Fundraisers
Bidding for Hope! Join the 2025 Cure GM1 Conference Auction
Support our mission while having fun with your family! We have incredible options including LuxGive vacations to Hawaii, Mexico, Disney, and more. Every dollar raised goes directly toward GM1 research and helping families attend our conference free of charge.
Online bidding starts July 8th at this link.
Host your own!
Want to host your own fundraiser? Visit our Fundraising Ideas page and check our guide to help you organize a fundraising.
Learn all about fundraising for Cure GM1
Angelversaries and Birthdays
Ways to Give – Your Support Matters
RaiseRight | iGive | Charity Miles | 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