Cure GM1 Catalyst
COMMUNITY ENGAGEMENT
ADVOCACY
BIOTECH NEWS
FUNDRAISERS
ANGELVERSARY & BIRTHDAYS
THANK YOU! It is with immense gratitude to announce that together we have raised $102,057 as part of the Cure GM1 Foundation’s end-of-year giving campaign. This includes a fully maxed-out match of $30,000, which this Cure GM1 community turned into $60,000. We are so grateful for your trust and generosity through this challenging fight for life.
Thanks to your support, we will keep pushing to advance potential therapies and treatments for these precious children. This includes continued work supporting the promise of enzyme replacement therapy in improving outcomes for children living with GM1. It also means supporting greater data collection via our GM1 Census; advocating for greater awareness of GM1 among pharmaceutical and biotech companies; and fighting for more clinical trials relevant for our ultra-rare disease community.
It has been over a decade since Cure GM1 was approved by the federal government as a 501(c)(3) nonprofit. Although GM1 was first named as a distinct condition in 1968, the very first clinical trials for GM1 started in this decade following the founding of Cure GM1, demonstrating the critical role of advocacy is drawing attention and resources to GM1 gangliosidosis.
This holiday season, our wish for the holidays remains the same, a proven, effective, and accessible treatment for all those dying due to GM1. The holidays can be complex for families missing their loved ones, those who are hospitalized, and those who can’t participate in the festivities due to health concerns. Nonetheless, we extend our warmest wishes to everyone in this community.
May the holidays be restful and may 2026 bring continued progress and hope.
Community Engagement
Kinley and Kennedy’s Story
Sisters Kinley, 12, and Kennedy, 9, were both diagnosed with GM1 gangliosidosis five years ago. In this story, their father shares how the disease is progressing in both of his daughters, and why he believes enzyme replacement therapy (ERT) holds promise for future children impacted by GM1 gangliosidosis. “Though it can’t reverse the damage that has been done, the prospect of early detection paired with ERT is very exciting. In fact, we have examples of ERT for other diseases that have made monumental changes in people’s lives – going from an outlook of early death and dependence to one of getting married, graduating college, and living a ‘normal’ life.”
December Community Chat – December 18, 7 p.m. ET/4 p.m. PT
We hope to see you at our December 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.
Sign Up for the Clinical Research ID (CRID)
The Cure GM1 Foundation encourages families to sign up for the CRID, a patient-generated identifier system designed for use in clinical trials. The CRID reduces survey fatigue by eliminating the need to supply (and resupply) personally identifying information. Additionally, it helps families gain more visibility into the research studies in which they’re enrolled.
On the research side, the CRID allows data identifiers to be reused, merged, and even added to studies retrospectively, increasing the amount of data available. This project is free for parents and patients. To learn more, click here.
Advocacy
Mark Your Calendars!
Make a plan to spread awareness for GM1 gangliosidosis this coming year! Below, we share a list of key advocacy dates for the first half of 2026.
February 28, 2026: Rare Disease Day. Download this information packet. You’ll find information on getting involved on social media, organizing an event in your area or reaching out to your local news media.
February 24-26, 2026: Rare Disease Week. Registration for this coming year’s events in Washington, D.C., opens January 7, 2026. For an agenda of 2026 activities, click here.
February 2-6, 2026: 22nd Annual WORLDSymposium. An annual research conference dedicated to lysosomal disease. San Diego, California. Learn more here. Christine will be attending and presenting a poster on GM1.
May 23, 2026: GM1 Day. Help spread awareness of GM1 gangliosidosis. Simply download our free action kit, which includes a social media profile frame, logos and more here.
Keep up with us and check out our annual calendar for other important dates to remember.
Pediatric Priority Review Voucher (PRV) Renewal
Great news! This week, the House of Representatives voted to reauthorize this special program to incentivize rare disease drug development. Now, we need the Senate to follow suit. Help reignite support for the Give Kids a Chance Act of 2025 (H.R. 1262/ S. 932) so this critical incentive program continues. Sign on here.
Access to Genetic Counselor Services Act Reintroduced
The Access to Genetic Counselor Services Act (HR 6208) has been reintroduced in the U.S. House of Representatives. The bill would allow genetic counselors to be recognized as Medicare providers. This would expand patient access to genetic counseling and reduce delays in both testing and treatment decisions. It is expected that the bill will be introduced to the Senate next week. Click here to ask your Congressional representatives to support HR 6208.
Cure GM1 Signs Letter in Support of a Strong and Predictable FDA
It’s true that research and the biotech industry are inherently volatile. Unfortunately, the instability has increased due to leadership changes and staff departures at the FDA. This letter encourages FDA to aim for increased stability to maintain its critically important leadership position, benefiting the entire healthcare ecosystem. To join us in signing the statement, click here.
Biotech and research News
Azafaros Trial Sites Are Opening
The Azafaros NAVIGATE study continues to open new sites. As a reminder, NAVIGATE is a double-blind, Phase 3 study to assess an investigational medication called nizubaglustat for the treatment of Niemann-Pick type C disease and the GM1 and GM2 gangliosidoses. The following locations are now open: Portugal, Spain, California, Paris, Sweden. Many more sites are being added. To see the information on the sites, you can see the study on clinicaltrials.gov.
FDA’s New Plausible Mechanism Pathway
FDA announced a new pathway for personalized medicines where randomized placebo-controlled trials are not feasible. This new approach emphasizes biologic plausibility and mechanistic rationale over population-level evidence.
Click here to read the article.
Click here to read an article in the New England Journal of Medicine
Fundraisers
Thank you, Harrison and Law Families!
We would like to express our gratitude to the Harrison family for their ongoing support and commitment to our critically important cause. For our year-end Giving Tuesday campaign, they increased their initial goal from $10K to $15K.
We hope you will read the Harrison family’s updated story above. It reflects back on the five years since receiving a GM1 diagnosis. And check out their video in support of ERT here.
Additionally, we would like to thank the Law family for their continued support, as well as their sharing Violet’s legacy in support of enzyme replacement therapy. See Violet’s video here.
Raise Awareness of GM1 With Your End-of-Year Gift!
For every gift of $50 or more to the Cure GM1 Foundation through December 31, we will send you a free magnet so you can show your support for those living with GM1! Click to give now!
Support Cure GM1 for the Holidays
Did you know you can support the Cure GM1 Foundation while you shop? Check out the following opportunities.
Flower Power Fundraising Holiday Sales: The sale includes amaryllis and paperwhite flowers and holiday decorations, with each purchase earning 40% for Cure GM1. Now through Dec. 15!
Minted.com: Get discounts greeting cards, prints, and more. Enjoy a 20% discount, and Minted will donate 10% of net sales. Use code: FUNDRAISECGM1
RaiseRight has many gift card offerings and a new online shopping program where purchases can generate donations to Cure GM1. Don’t miss out! It’s an easy way to give during the holidays and to benefit Cure GM1!
Holiday Gear and Merch to Support Cure GM1
Angelversaries & Birthdays
The following is a list of December angelversaries and birthdays in the GM1 gangliosidosis community. We deeply apologize if we ever miss a date or birthday. Please always feel free to share updates and feedback.
Annual Candlelight Memorial
Please join us to observe the Compassionate Friends Worldwide Candlelight Ceremony on December 14 at 7 p.m. your time. Share photos and candles.
Ways to Give – Your Support Matters
RaiseRight | Walmart Spark Good | 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