Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS
A note from Christine
Dear GM1 Community,
What an incredible month August was! Our 2025 International GM1 Community Conference brought together participants from around the world. We were excited to hear from Azafaros that the first trial participant has started taking the treatment. We witnessed the power of our shared hope, courage, and commitment.
As we move into September, I’m thrilled to announce the launch of our new Do It for GM1 campaign—a movement where anyone can join by fundraising through activities they love. Whether it’s running a marathon, hosting a bake sale, or organizing a community event, every effort matters in advancing our efforts to find a treatment for GM1.
This month marks my birthday. I have a birthday fundraiser in honor of my daughter Iris whose light continues to inspire my work every day. Her story reminds us why we fight—not just for today’s families, but for every child who will face this journey in the future.
The progress we celebrated at our conference—from an upcoming fetal gene therapy study to a new substrate reduction trial—all exciting progress in our fight for meaningful change. Together, we’re not just advancing science; we’re building hope, connection, and a future where no family faces GM1 alone.
Christine Waggoner
Community Engagement
Tiffany’s Story
Meet Tiffany, a young woman with a bright smile who was diagnosed with GM1 gangliosidosis at a young age and was given 5 years to live. Despite losing her ability to walk and talk, she remains a happy and strong young woman at age 23 who has taught her parents to find joy in simple pleasures and appreciate love expressed through her eyes. Her story shows the resilience of children with GM1 and why they inspire and transform the lives of those around them. See Tiffany’s Story
2025 International GM1 Community Conference
Our annual conference exceeded all expectations, bringing together families, researchers, and families. With 133 registered individuals including 103 GM1 family members from 20 countries, we truly embodied our international community spirit.
Conference highlights:
At the GM1 Family Speaker Series, families share their deeply personal journeys. Deepika, honoring her son Niamh, reminded us that “love and happiness live in life’s quiet moments—his laughter, his sister’s companionship, his little hands holding ours. Though his disease took so much, it never took the love that surrounded him.” Megan shared Ben’s story—diagnosed with juvenile GM1 on December 22, 2023, after years of unexplained symptoms. When the diagnosis finally came, it was the first case his doctors had seen. Guadalupe, speaking about Eva and Franco from Buenos Aires, expressed hope for “faster diagnoses, greater awareness of rare diseases, and future treatments.”.
Keynote speaker Amanda Griffith Atkins provided profound insights on navigating the emotional journey of caregiving, acknowledging that “grief recurs throughout the journey” and urged caregivers to “honor their whole selves, embrace complex emotions, and find meaning amid hardship.” Her most powerful message: “Love and grief can—and do—coexist.”
Mary McDirmid shared essential special needs planning guidance, reminding families to “stay present, plan ahead, and know you are not alone on this journey.”
Research Breakthroughs: Dr. Caroline Hastings presented encouraging updates from the NAVIGATE phase 3 trial, while Billie Lianoglou shared plans for a fetal GM1 gene therapy trial. GEMMA Biotherapeutic’s Monique Molloy outlined their mission to expand global access to gene therapy, and Dr. Gouri Yogalingam presented our foundation-led enzyme replacement therapy program.
Here are some photos of the Conference. Our children had their share of fun too!
View the Photo Album
Conference recordings are now available to registered attendees. If you haven’t received the link, please reach out to us at info@curegm1.org. Please share your photos with us and complete our feedback survey to help improve next year’s event: https://form.jotform.com/242578987853176
Join the New GM1 Census Now
We have a very important initiative that will be the reference for all future research on GM1. This census will help us better understand our community’s needs, track disease progression patterns, and provide crucial data for researchers and pharmaceutical partners. Your participation is vital in building knowledge about GM1 and showing the world that our community exists. It is absolutely essential to attract the attention of investors and biotechs to demonstrate that our community is research-ready. Join the census today
UCSF Fetal Gene Therapy Survey
UCSF is preparing for a groundbreaking first-in-human phase 1 clinical trial of prenatal gene therapy for GM1 gangliosidosis. This pioneering approach aims to prevent irreversible organ and brain damage before birth, potentially transforming outcomes for our youngest patients.
The research team wants to understand your perspectives on this potential study and learn about your experiences with pregnancy planning. Your input will help shape this revolutionary approach to treating GM1 at its earliest stages.Share your perspective: https://redcap.ucsf.edu/surveys/?s=LEDRWK7AJXHAMLNX
Advocacy
September Awareness: Leukodystrophy and Newborn Screening
Leukodystrophy Awareness Month: GM1 gangliosidosis is sometimes grouped with leukodystrophies, but is classified as a leukencephalopathy—a condition affecting the white matter of the brain. Understanding this classification helps connect our community with broader rare disease advocacy efforts and research initiatives targeting similar neurological conditions, amplifying our collective voice for increased awareness and research funding.
Newborn Screening Awareness Month highlights the critical importance of early detection. Though GM1 is not yet included in standard screening panels, promising research methods are rapidly advancing. We currently have two cases of pregnant women where GM1 has been detected—a powerful demonstration of how screening can open doors to life-saving prenatal interventions. These real cases make the need for expanded screening programs more urgent than ever.
Biotech News
Azafaros NAVIGATE Trial Update
At our conference, it was announced that the first participant has been enrolled in Portugal, marking a significant milestone. New sites will be opening up to expand enrollment opportunities for families worldwide. For more details on the NAVIGATE trial, watch Azafaros’ presentation at our conference
Fundraisers
Introducing Do It for GM1: Turn Your Passion Into GM1 Research
We redesigned our Move for GM1 to a more inclusive campaign called “Do It for GM1”—where anyone can make a difference by fundraising through activities they love. Whether you’re running a marathon, hosting a dinner party, organizing a garage sale, or planning a community event, every effort contributes to advancing life-changing treatments.
Why it matters: Fundraising isn’t just about raising money—it builds hope, connection, and joy through shared causes with loved ones. Whether you’re planning something big or starting small, every fundraiser makes a difference in the fight against GM1.Your fundraising enables research that brings hope to families worldwide.
How It Works:
- Choose an activity you’re passionate about
- Share your story and connect it to GM1
- Rally your community to support your efforts
- Share our GiveButter fundraising page (https://givebutter.com/iXRo17)
- Celebrate your impact on GM1 research
Our team is happy to discuss ideas and offer you support. Reach out to us – info@curegm1.org.
Upcoming Fundraising Events:
Year-End Giving Campaign – December 2025
Giving Tuesday – November 2025
Let everyone know that you’re doing it for GM1 this September. At the link below, you can create your next Facebook profile picture with our own Do It for GM1. Download the Frame!
Show your support with our Do It for GM1 T-shirt. The shirt comes in all sizes and in four colors! Click below to visit the online store.
Visit the Cure GM1 Store
Angelversaries and Birthdays
Ways to Give – Your Support Matters
RaiseRight | Walmart Spark Good | 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