Cure GM1 Catalyst
Happy March!
First, we want to thank you for your support in making Rare Disease Day and Rare Disease Month 2026 so memorable! Together, we were able to raise awareness of GM1 gangliosidosis and other rare diseases all over the world. The more we share, the closer we are to finding treatment. I want to especially thank all the GM1 families who took the time to advocate and share their personal stories with the world and on the news. Together, we are helping increase awareness and understanding. (In case you missed it, check out this beautiful piece from GM1 parent Ashley Thornton, as well as the video below about Kiaan’s GM1 journey.
I also want to thank all of you who donated to Iris’ 18th Birthday Fundraiser. With your help, she has built a beautiful legacy of hope for those living with GM1. Click below to view a piece about Iris that recently aired at our local television station.
And, I can’t forget Cure GM1’s presentation at the WORLD Symposium in San Diego. You can check out our full poster and photos below.
Looking ahead, we’re setting our sights on spring, which is an excellent time to brainstorm activities for GM1 Day on May 23! If you are interested in requesting a proclamation in your state, please let us know. You can email jess@curegm1.org to get all the details.
We continue to push forward on our enzyme replacement therapy project and more. Every bit of help makes a true difference. Thank you again for your support. Together, we’re making a difference.
Community Engagement
Save the Date! The Annual GM1 Community Conference is Oct. 2, 2026
This year’s event is 100% virtual. While we are still waiting on full agenda and speaker information, registration is now open and free for GM1 families. Click here to visit our conference page, which includes a registration link, sponsor information and more.
Daxton’s Story
“GM1 took over our lives long before it ever had a name. Little by little, it began taking pieces of our boy away, even as we poured everything into helping him.“ Click to read Daxton’s story, written by his mother.
Congratulations to our Rare Disease Day Photo Contest Winner: Imogen!
“Imogen is our beautiful 9-year-old daughter, diagnosed with GM1 Type 2 at the age of 2. She adores her siblings, Naomi and William, and they adore her. They’re always doing things together. She is an amazing girl and such an important little person in our family!” – Imogen’s mom
And thank you to all of you who sent in your beautiful submissions for this year’s contest!
March GM1 Community Chat: Thursday, March 19, 1 p.m. PT / 4 p.m. ET
Don’t miss our monthly community chat Thursday, March 19 at 1 p.m. Pacific / 4 p.m. Eastern. This is an opportunity to connect with other GM1 caregivers in a safe and supportive environment. For community privacy, registration is required. Click here to register now.
Advocacy
Free Summit: Empowering Patients 2026
The American Society of Gene & Cell Therapy is offering a free virtual summit titled, “Empowering Patients 2026: A Cell and Gene Therapies Summit.” The event will be held March 11-12, 2026, from 10 a.m. to 1 p.m. (Eastern time). The summit was designed for patients, caregivers, advocates and researchers. To register, click here.
Act for Ultra Rare Webinar on Legislative Policy Now Available
The event, held Jan. 30, was titled “Legislative & Policy Updates: Ultra-Rare Disease Legislation in the 119th Congress.” The event featured numerous speakers and stories. Click here to review a recording of the event, including Q&As.
Spring 2026 Young Adult Rare Representatives Leadership Academy Opening Soon
The EveryLife Foundation for Rare Diseases announced that its YARR program will launch its 2026 cohort soon. The program, which focuses on building advocacy skills in public policy, drug-development and regulatory issues, is open to those ages 16 to 30 living in the United States. Applications are open now through March 10. All applicants will receive a decision by early April. For more details, click here.
FDA Hosted Virtual Public Meeting for Rare Diseases
The event, held Feb. 23, invited rare disease patients and advocates to share their treatment experience. Though the event has passed, you can still get involved via CURE ID, a free platform sharing novel uses of existing drugs as a means of identifying treatments for diseases that currently have no treatment options.
research & biotech news
Azafaros Publishes Promising Data in Nizubaglustat Trial
Azafaros recently published data regarding nizubaglustat’s efficacy in treating patients with GM2. Results appeared in the Jan. 7 issue of the Journal of Inherited Metabolic Disease.
The Phase 3 study is evaluating the effectiveness of nizubaglustat in late-infantile and juvenile forms of Niemann-Pick Type C and late-infantile and juvenile-onset GM1 and GM2. U.S. locations now recruiting include Fairfax, Virginia, and Oakland, California. For a full list of locations where the trial is open, click here.
Sanofi’s Venglustat Shows Promising Outcomes in Phase 3 Study of Type 3 Gaucher Disease
This is significant news for the GM1 community. In a previous venglustat study focused primarily on late-onset GM2 patients, GM1 patients were given venglustat as participants in the secondary basket trial group. The study did not produce the intended results for late-onset GM2 patients and was eventually closed. However, venglustat did show improvements for GM1 patients, as mentioned in this publication. A new phase 3 study of venglustat for Gaucher disease (another lysosomal storage disease) demonstrated efficacy on all primary outcome measures. The success in the Gaucher trial means that the company will proceed with applying for approval of venglustat for Gaucher disease. Click here to read the full press release.
AAV9 GM1 Study Featured in New England Journal of Medicine
The New England Journal of Medicine recently shared peer-reviewed results of an AAV9 gene therapy study focused on patients with GM1 Types 2a and 2b. Primarily, the researchers focused on patient safety, followed by changes in cerebrospinal fluid GM1 ganglioside concentration and other biomarkers. Click here to read the full article, which showed that patients did see some improvement.
Cure GM1 Presents at WORLD Symposium
At this year’s WORLD Symposium, Cure GM1 President and Co-Founder Christine Waggoner presented a poster titled, “Global Characterization of GM1 Gangliosidosis: Insights from a Patient-Powered Registry.” The study used Cure GM1 registry insights from patients in 36 countries and six continents. Among other insights, the study found that GM1 type distribution varied significantly between the United States and other parts of the world. It also found a heterogeneity in onset symptoms. Click here to view the poster.
FDA Shares Framework for Accelerating Treatment Advancement for Ultra-Rare Diseases
The U.S. Food and Drug Administration recently issued preliminary guidelines that would make it easier to develop treatments for those with ultra-rare diseases. The guidance is meant to make it easier to advance drug development in cases where small patient populations make randomized, controlled trials unfeasible and where pharmaceutical companies have traditionally considered drug development unprofitable. The announcement specifically mentions gene editing, but it could also be used for other drugs and treatments. Click here for the full release.
Dr. Simon Jones transitions to Denali Therapeutics
Dr. Jones, a scientific and clinical advisor to Cure GM1, transitioned to a new role in biotech as an executive medical director at Denali Therapeutics. In the past, amongst many other roles, Dr. Jones was a principal investigator studying GM1 in several clinical trials in the UK. Click here to learn more.
Fundraisers
Thank you, Hough Family!
A special thank you to the Hough family, whose friends organized a fundraiser in honor of Aidan’s heavenly birthday. Aidan would have been 5 years old.
Help make a difference by planning a birthday or special occasion fundraiser this year. Email jess@curegm1.org to have a personalized giving page created for your child.
Time for Easter Chocolates!
Get a jump on your Easter treats while supporting the Cure GM1 Foundation. Click here to get started.
Roses are Red, Violets are Blue – Your Next Flower Order Could Help Cure GM1, Too!
Need to order a bouquet of flowers? You can do so while supporting the Cure GM1 Foundation. Click here and enter your delivery date to check out availability.
And Don’t Forget the GM1 Store
It’s your one-stop shop for everything you need to show your support for those living with GM1. Click here to shop now.
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