Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS
A note from Christine
Dear GM1 Community,
What an exciting month ahead!
Our annual conference is just three days away on August 8th, and we can’t wait to connect with families from around the world. We’re thrilled to offer live translation services this year, ensuring our global community can fully participate. Don’t miss our keynote presentation, “How to Handle More Than You Can Handle: A guide to navigating the emotional experience of caring for and raising disabled children with high-support needs,” along with the latest updates on our ERT project progress – and all the advancements and updates that bring us closer to treatments.
This month also brings wonderful news as we welcome Gouri Yogalingam-Crommie to our Board of Directors and Megan Reindl as our new Community Giving Officer. But perhaps most importantly, we’re launching our Do It For GM1 campaign at a critical moment. Our ERT research is showing extraordinary promise, but we need your help to fund the next crucial phase. Every fundraiser brings us closer to the treatment our children desperately need.
The momentum is building, and hope is growing stronger every day. Together, we’re making a difference through community, research and advocay.
With gratitude and determination,
-Christine Waggoner
Community Engagement
Solomija’s Story of Courage
This month, we share the story of Solomija, a young woman who faced GM1 with extraordinary courage and dignity. Her friend describes how Solomija approached her diagnosis as “a battle,” while navigating the realities of this devastating disease. Her story reminds us why our work matters and honors all those we’ve lost to GM1. Read Here
Remembering Gael
It is with heavy hearts that we share the passing of precious Gael on July 23, 2025, at just one and a half years old. Diagnosed with Type 1 infantile GM1, Gael showed remarkable resilience throughout his brave fight. Gael’s legacy strengthens our mission to find treatments so other babies can have a chance at longer, healthier lives. We hold Gael’s family in our hearts. Read Gael’s Story
Welcome Our New Board Member!
Gouri Yogalingam-Crommie has joined our Board of Directors, transitioning from her role as Scientific Advisor. As Founder and CEO of Pyra Bio and former Director of External Collaborations at BioMarin, Gouri brings 12+ years of experience developing therapies for lysosomal storage disorders, including GM1 gangliosidosis. Her expertise spans from academic research to three successful drug approvals, making her an invaluable addition to our leadership team.
Introducing Our Community Giving Officer
We’re pleased to welcome Megan Reindl as our new Community Giving Officer. She is the mother of 10-year-old Ben, who was diagnosed with juvenile GM1 Type 2b in December 2023. Her passion for finding a cure and supporting other families drives her commitment to this role. We’re grateful to have her dedication and perspective as part of our team.
Cure GM1 Community Chat
Thank you to those who joined us for our call in July. These calls are an effort to bring our community closer together, share information, ask questions, and talk about the challenges and joys of caring for someone with GM1. Bereaved parents are more than welcome, too. We dedicate this space to all of our community members.
Our next meeting will be on September 11, 9 am PT / 12 pm ET. Register Here
Advocacy
Major Health Insurance Changes Affect Rare Disease Families
The recently signed “Big Beautiful Bill” will significantly impact healthcare access for rare disease patients. An estimated 16 million people may lose insurance coverage due to changes in Medicaid requirements and Affordable Care Act marketplace plans. These changes include new copays, work requirements, and elimination of premium tax credits that many families rely on. Watch EveryLife Foundation’s webinar unpacking what it means for our rare disease community, and what you can do moving forward.
Newborn Screening Updates
The House Energy and Commerce Committee recently held hearings on the Newborn Screening Saves Lives Reauthorization Act. Early detection through newborn screening is critical for GM1 patients.
Watch the Hearing Recording Here
Biotech News
New Webinar Available: Azafaros Navigate Study Update
We’ve published our recent webinar featuring Azafaros and their Phase 3 Navigate study for nizubaglustat. This important session provides updates on this potential GM1 treatment currently in clinical trials. The webinar offers valuable insights for families considering participation or wanting to stay informed about emerging therapies. Watch the Azafaros webinar on YouTube
NIH Research Study – Your Voice Can Help Advance AI in Genetic Medicine
The National Human Genome Research Institute (NHGRI) at NIH is conducting an important study on how artificial intelligence models can better identify genetic conditions like GM1. They’re seeking parents and caregivers of children with diagnosed genetic conditions to participate in 30-60 minute interviews via video or phone. All participants must be 18 or older, and the research team will provide full study details and answer questions before seeking your consent. The research aims to improve AI tools that may become part of future healthcare, potentially helping with faster diagnosis and better management of genetic conditions.
Interested in participating? Contact: medicalgenomicsunit@nih.gov
Fundraisers
Join Our Do It For GM1 Campaign – Help Fund Life-Saving ERT Research
Every moment counts in the race to bring enzyme replacement therapy (ERT) to GM1 families. Our groundbreaking ERT research is showing incredible promise, but we need YOUR help to reach IND.
Do It For GM1 is a movement of GM1 families and friends. Whether you run, walk, bake, or craft, every action brings us closer to a treatment that could transform our lives. We’re funding the research that pharmaceutical companies won’t prioritize, making breakthroughs that could benefit every person living with GM1.
Why This Matters Now:
- Our ERT research is at a critical funding stage
- Every dollar directly supports drug development
- Your participation amplifies our reach and impact
- Together, we can accelerate the timeline to treatment
Ready to make a difference? Start your own fundraiser or join an existing event. No effort is too small when it comes to saving the lives of our children. Launch Your Do It For GM1 Campaign
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
Bidding for Hope! Join Our 2025 Conference Auction
Don’t miss your chance to bid on amazing items while supporting GM1 research! Our annual conference auction features unique travel experiences donated by our generous community. Every bid directly funds our new Enzyme Replacement Therapy project and brings us closer to finding a life-saving treatment.
Special Conference Offer:
Take your family on a behind-the-scenes tour at Pixar Animation Studios, where Toy Story and your favorite animated films come to life! Up to 8 guests – a once-in-a-lifetime experience.
ATTENTION: Bidding closes August 9th at 5:00 AM EDT.
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