October 2025 Cure GM1 Catalyst

Cure GM1 Catalyst

COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS

Let’s take a minute to celebrate Christine, Connie, Niclas, and Kevin for “Doing It for GM1.” Their combined efforts have raised over $35K! The month closed with great news from SylamoreBio, who received a $3 million NIH grant to advance their innovative GM1 research.

This is real progress! 🎉

As we move into October, we’d like to call you to join an important initiative —the GM1 Census, a critical tool in drawing more attention to GM1. We’re also sharing a new video highlighting our conference and the incredible GM1 community.

Don’t miss all the updates and stories below!

Halloween Smiles 🎃

We’re celebrating our incredible GM1 kids this Halloween season! These little superheroes, princesses, and pumpkins bring us so much joy. Send us your 2025 pics!

Community Engagement

Watch the Two Videos We Released!

Iris’s Story: How ERT Can Save GM1 Children

Watch Iris’ story and learn how Enzyme Replacement Therapy can be a new game changing tool in treating GM1. If you donate to this project, you’ll be giving Iris and every child with GM1 the chance at a better life.

Donate Today

2025 GM1 Conference Highlights

Our August conference was powerful, full of connection and hope! The video captures highlight moments that will make you smile along with our incredible community. These gatherings remind us of the strength we find in unity and shared purpose.

Nella’s Story with GM1

Nella became the first child enrolled in a small molecule clinical trial, and today, at eleven years old, she is thriving—attending school, making friends, and participating in weekly activities. However, in late 2024, her clinical trial was shut down, despite success in pediatric cases. There’s no regulatory approval or certainty that what’s helping Nella today will be available tomorrow.

We fight tirelessly to secure clinical trial approvals that can save GM1 children, and we cannot afford to lose any ground. We must stand up for all GM1 children to ensure safe access to treatments!
Learn more

Aidan’s Legacy: Honoring a Beautiful Life

Aidan lived on this earth for just 206 weeks before passing away last February. No parent or grandparent should endure the pain of losing a child. Connie, Aidan’s grandmother, has shared her beautiful testimony about her time with him. She keeps his memory alive and honors him every day, ensuring his legacy continues to inspire our fight against GM1.

Read Aidan’s story by his grandma

Remembering Harrison

September last year we lost Harrison. We want to share the beautiful words of his father Ken, who shared a powerful tribute reflecting on this first year of grief and remembrance:

“Harrison, you have been in my heart for a year where I wasn’t sure how I would make it. You’ve brought me joy and your loss has given me great sorrow… Not a day will go by without you in my mind and you will be with me forever. You were the best of us!
Read the full tribute on Facebook

October Community Chat – October 24th

Don’t miss our October Community Chat, a safe space to share experiences and support one another. These monthly gatherings provide invaluable emotional support and practical advice from families who truly understand the GM1 journey.

Register Here

Join the New GM1 Census

By joining the GM1 census, we demonstrate to researchers and drug developers that we are numerous and worth investing time and resources in. All information is protected and anonymized—you cannot be identified individually. If you have questions, reach out to us at info@curegm1.org.

Join today

New Annual Event Calendar Available

Stay connected with our community and mark your calendars for important dates so you never miss an opportunity to engage with our community!

View Calendar

We’re Excited to Welcome David Whiteman as Clinical Advisor

For both our data sharing initiative and Enzyme Replacement Therapy (ERT) project, Dr. Whiteman will be serving as our Clinical Advisor. His expertise will be invaluable in advancing our clinical research efforts and ensuring families have access to the most current treatment options and clinical trial opportunities.

Advocacy

RISE Workshop FDA Proposal Cure GM1 is leading a coalition of patient organizations, research groups, and biotechs in urging the FDA to host a RISE Workshop that focus on primary disease biomarkers for neurodegenerative lysosomal diseases, potentially accelerating approval pathways for life-saving therapies. Families shouldn’t have to wait decades for treatments when biomarkers can demonstrate real disease activity.

Read the Proposal

Give Kids a Chance Act – PRV Program Renewal The House Committee on Energy and Commerce advanced the Give Kids a Chance Act, which would reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) Program for five years. This market-based incentive has already brought therapies to children with 40+ rare diseases. This program provides critical incentives for companies to develop treatments without costing taxpayers

Take Action and Support PRV Reauthorization | Visit the NORD Action Page

Rare Disease Week 2026 – Travel Reimbursements Available Applications are now open for travel reimbursements to attend Rare Disease Week on Capitol Hill (February 24-26, 2026). This is a crucial opportunity for patients to advocate directly with Members of Congress for rare disease research and policy support. Application closes November 8th.

Apply Here

Biotech News

SylamoreBio Receives $3 Million NIH Grant SylamoreBio has been awarded a significant NIH grant to advance GM1 Gangliosidosis research using their innovative SyLEC delivery system. This approach aims to deliver beta-galactosidase enzymes to the brain through non-invasive administration.

“This novel approach has the potential to treat brain pathologies through non-invasive administration. By eliminating the need for gene transfer into central nervous system tissue, this method offers significant safety advantages,” said Dr. Walter Acosta, Chief Scientific Officer.

New Clinical Trial Resource from Azafaros Azafaros has launched a clinical trial navigation website to help GM1 families find and understand available trials. This valuable resource connects families with ongoing research opportunities.

Explore Trial Website

Fundraisers

Do It for GM1 Campaign Results

Thank you so much to Connie, Christine, Niclas, and Kevin for Doing It For GM1 this September! A huge thank you to everyone who supported their efforts through donations—this is true recognition of their dedication.

Together we raised over $35K that will directly support GM1 research, drug development and advocacy!

Support the Campaign