Don’t miss what is happening in our community and the broader rare disease community! There are many important opportunities to engage and to make a difference! Read on to learn more on continued progress and help build momentum.
Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS
It’s GM1 Awareness Month: Amplify Your Voice
Dear Friends and Supporters,
As May begins, we’re filled with anticipation for GM1 Awareness Day on May 23rd. This is a month-long effort to shine a light on GM1 gangliosidosis and the remarkable community that surrounds it.
We’ve submitted 12 proclamation requests to officials across the country. While these official recognitions help legitimize our cause, the true power of awareness comes from you—our families, caregivers, and supporters who live with GM1 every day.
Why Your Story Matters
Personal stories create connections that facts alone cannot. When you share your GM1 journey with local media, you transform a rare disease into a human experience that resonates. This can help healthcare providers recognize symptoms in undiagnosed patients, inspire researchers, and show isolated families they’re not alone. If sharing feels intimidating, resources are available from Global Genes, WiRare, and EveryLife Foundation.
Advancing Hope: Our ERT Project
We’re excited about our Enzyme Replacement Therapy (ERT) project, which addresses GM1’s underlying cause by replacing the missing β-galactosidase enzyme. Learn more at www.curegm1.org/ert-project.
In this issue, you’ll find updates on GM1 Awareness Day initiatives, research developments, and community stories. I hope you’ll find the reading helpful.
Community Engagement
In Loving Memory of
Joaquim, Izaac, Timothy, Ela
It is with heavy hearts that we acknowledge the passing of four precious members of our GM1 community this April: Joaquim, Ela, Izaac, and Timothy. Three of these beautiful children—Joaquim, Izaac, and Timosha—left us just this past week. Each of these children embodied strength, resilience, and love in the face of unimaginable challenges.
As a community that understands this painful journey, we stand together in solidarity with their families. Their lives, though too brief, continue to inspire our mission and strengthen our resolve to accelerate research toward treatments
“Joaquim was strong, a little warrior fighting to live. We are certain that during his short time with us, he was a happy baby. Now he is a little angel in heaven. We will feel great longing and emptiness in our home and in our hearts.“
“Izaac was the light of everyone’s life. He touched so many people and loved everyone. He filled the room with smiles wherever he went. He always surprised everyone with his gorgeous eyelashes and hair. He meant the world to me and will be missed by so many.”
“Timosha, the meaning of the name is ‘respecting God’. Our space boy. I love you to the moon and back.”
“Some souls shine so brightly that even after they’ve left us, their light continues to guide our way.”
May is GM1 Awareness Month!
From Rare to Recognized: Make GM1 Known on May 23rd
Every story shared about GM1 gangliosidosis can reach healthcare providers, researchers, policymakers, and families searching for answers. As GM1 Awareness Day approaches on May 23rd, we’re calling on our community to amplify our voice by sharing about GM1 gangliosidosis with local media.
Local news outlets are trusted voices in communities across the country. When they share GM1 stories, they reach people who might otherwise never learn about this rare condition. One local news story can lead to earlier diagnosis for a child, inspire a medical student’s research path, or connect isolated families to vital support.
We’ve Made It Easy for You and prepared everything you need to approach your local media:
- Ready-to-use email templates for contacting editors
- Press release about GM1 Awareness Day
- Key talking points to use with local media
How to Participate:
- Email tayara@curegm1.org with your city and state
- We’ll help identify local media contacts and provide all necessary materials
Join us in turning May 23rd into a powerful day of awareness by sharing your GM1 journey with your community. Together, we can ensure that no family faces GM1 alone.
Join Our GM1 Community Chat on May 15
We invite all GM1 parents and caregivers to join our casual community chat on Wednesday, May 15th for an open discussion about anything on your mind. This is your space to:
- Ask questions about GM1 management, resources, or research
- Share your experiences, challenges, and triumphs
- Connect with others who truly understand your journey
When: Wednesday, May 15th, 9:00 AM PT (12:00 PM Eastern Time)
Where: Zoom – Register Here (Zoom’s translation feature will be available).
We hope to see you there!
Honor GM1 Dads This Father’s Day: Share Your Tributes
Father’s Day is approaching, and we want to recognize the incredible dads who demonstrate remarkable love and dedication every day in our GM1 community. We invite you to celebrate your own father, your partner, or a father figure who has made a difference in your GM1 journey.
How to Participate:
- Send us a photo of a GM1 dad/father figure with his child/children
- Include a meaningful quote or share a brief memory if you wish
- Email your submissions to tayara@curegm1.org by June 1st
We’ll create a special Father’s Day tribute featuring these photos and messages to honor the fathers who are true heroes in our community.
Gene Therapy Experience Wanted: Share Your Story!
Have you or a loved one undergone gene therapy? The Pediatric Gene Therapy & Medical Ethics (PGTME) group seeks your valuable insights to help shape future treatment approaches. Your feedback can directly influence how gene therapies are developed, administered, and evaluated—potentially improving outcomes for future GM1 patients. Share your experience today and make a difference.
Register Now: 2025 International GM1 Community Conference
Our Annual GM1 Conference this year will be a hybrid event taking place in Irvine, California. While virtual attendance is possible, in-person participation is highly encouraged to maximize the benefits of connection and community. The conference begins with a welcome dinner on August 7th, followed by a full day of presentations, discussions, and community building on August 8th.
Special Offers & Support Available:
- Translation Services: High quality translation provided through the Wordly platform, ensuring everyone attending virtually around the globe can fully participate regardless of language barriers.
- GM1 families attend completely free of charge thanks to our generous sponsors.
- Free Disneyland Tickets: Limited numbers of tickets are available and will be distributed amongst interested in-person attendees on a first come first serve basis. Rules apply as printed on the pass.
- Travel Scholarships: Limited funding still available to help families with travel costs to attend in person. Mail info@curegm1.org to apply.
Questions? Contact us at info@curegm1.org
Advocacy Events and Opportunities
Cure GM1 SignS Support to NIH Funding Increase for Medical Research
Cure GM1 has endorsed the Ad Hoc Group for Medical Research funding recommendation for the National Institutes of Health (NIH). The coalition recommends Congress provide “at least $51.303 billion for the National Institutes of Health for fiscal year 2026,” representing a $4.222 billion or 9.0% increase over the FY 2024 funding level.
This increase would directly impact rare disease research, including GM1 Gangliosidosis studies. Higher budgets enable more grants, expanded research programs, and greater potential for breakthrough treatments. An available Q&A document explains how NIH investment drives economic growth, secures U.S. global leadership in medical research, and details the harmful impacts of funding delays.
Cure GM1 Joins Coalition for Ultra-Rare Disease Treatment Innovation
Christine Waggoner has joined a coalition of stakeholders addressing critical barriers in ultra-rare disease treatment development. This group unites advocates, policymakers, clinicians, and researchers to modernize outdated policies that have stalled innovation for the rarest conditions. Many ultra-rare diseases like GM1 gangliosidosis remain untreatable despite scientific advancements because current regulatory frameworks don’t accommodate these conditions’ unique challenges.
The campaign kicked off on Tuesday, April 29, at the Rayburn House Office Building on Capitol Hill in Washington, DC, raising awareness among policymakers, and building momentum and support for the initiative. Learn more about this initiative at actforultrarare.com
Protect Newborn Screening, Systems Under Threat
The federal newborn screening advisory committee was disbanded on April 3, threatening the system that identifies life-threatening conditions in 14,000 babies annually. Without this expert panel, we risk returning to inconsistent screening where wealthier states provide better testing than others—directly impacting conditions like GM1 where early diagnosis is crucial.
Take Action Now: Contact your representatives through the EveryLife Foundation’s portal and share your family’s story with policymakers.
How Education Budget Cuts May Impact Students with Disabilities
Recent Department of Education budget cuts could threaten vital services for children with disabilities, including those with GM1. While funding for special education is promised to continue, advocates warn that reduced federal support may lead to larger class sizes, fewer support staff, and limited access to crucial assistive technologies.
Biotech News
Cure GM1 Foundation to Be Represente at National Health Council Symposium
Don’t miss this update on Christine Waggoner’s invitation to speak at the prestigious 2025 Science of Patient Engagement Symposium in Washington DC—a critical opportunity to raise GM1 awareness among healthcare policy influencers and decision-makers. Visit the event page to learn more.
FDA Commissioner Announces New Pathway for Ultra-Rare Disease Treatments
This groundbreaking announcement could transform treatment access for the GM1 community—read how the FDA’s new “plausible mechanism” approval pathway might bypass traditional clinical trial requirements for ultra-rare conditions like GM1, potentially unlocking investment and innovation.
Florida’s Sunshine Genetics Act Advances Rare Disease Screening
Learn how Florida’s House Bill 907 could make the state a leader in newborn genetic screening for rare diseases, potentially including more lysosomal storage disorders like GM1—a vital development as federal oversight of newborn screening faces challenges.
Fundraisers
Check out our GM1 Day Merch!
Celebrate Mother’s Day and father’s day with Cure GM1
Flower Power Spring Sales ends on May 15
Don’t miss out on their colorful selection of garden flowers and hummingbirds garden mix. They also have gardening kits for children!
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