Cure GM1 Catalyst
Welcome to July!
Summer has a way of slowing things down, and we hope you are finding joy in relaxed routines, longer days, and more time with your precious families.
I’m pleased to introduce our coordinator for this year’s virtual International Cure GM1 Community Conference, Cleopatra Frazier. She’ll be here to answer all of your questions and help us create an impactful and meaningful experience for all of you Oct. 2. More about Cleopatra below.
Whether you are walking this road as a GM1 family or showing up as an ally for our community, we warmly invite you to join us for this year’s Do It for GM1 campaign, August 1–31. This year’s campaign will support our goal of developing Enzyme Replacement Therapy (ERT) as treatment for GM1. We must raise $600,000 by January 2027 to keep the ERT project moving forward. Your active support of Do It for GM1 could get us closer to that goal. The beauty of this campaign is that it truly meets you where you are. It doesn’t matter what you do: cooking, running, playing piano, traveling, doing puzzles. What matters is that you do it with heart. Together we can raise both awareness and critical funds for GM1 research. Check out the full story below.
Enjoy the summer, hold your loved ones close, and know that our entire Cure GM1 team is working hard to move our mission forward.
Community
Leonor’s Story
“Leonor has taught us that dignity and joy can be found in the smallest details: a touch, a glance, or a quiet moment of family togetherness.” -Leonor’s Mom
In 2019, we shared the story of Leonor, who was diagnosed with GM1 at age 9. Here, her mother shares how Leonor is doing seven years later.
Welcome, Cleopatra!
Cure GM1 welcomes Cleopatra Frazier as coordinator for this year’s International Cure GM1 Community Conference. Cleopatra has spent more than a decade coordinating speaker programs, conferences and community conventions for organizations including the Howard Hughes Medical Institute. We are so glad to have her on our team! Reach her at cleopatra@curegm1.org.
Don’t wait to register! Registration is free but required to attend! We are incredibly excited that new teams will be presenting research that has not been presented before for our GM1 community. We intend to make it the most impactful year yet!
Discount Code Now Available for Biotech Industry and Friends!
For a limited time, use discount code EARLYBIRD2026 to receive a 20% discount for our virtual 2026 International Cure GM1 Community Conference Oct. 2. The discount is good for both Biotech Industry and Nonprofit/Academia/Friends/Research passes.
Click here to register now.
A Heartfelt Thank-you to Kylie Harrison
We are sending love and gratitude to Kylie Harrison, who has recently stepped down from the Cure GM1 Foundation’s Board of Directors after five meaningful years of service. As a GM1 parent, Kylie’s perspective has been critical in guiding the organization in its efforts to fund research for this cruel disease. Please join us in thanking her for her dedicated service. (See Kylie’s Cure GM1 board welcome photo from 2021!)
Join our Cure GM1 Community Chat Thursday, July 23, at 2 pM PDT / 5 pM EDT
We have recently seen an uptick in interest in our monthly community chats, and we are so grateful to all of you who have chosen to attend, share your perspective and connect with other families who may be newer to this disease.
We hope you join us for our next meeting. To respect the privacy of all community members, registration is required.
When: Thursday, July 23, 2 p.m. PDT/5 p.m. EDT
Where: Online (Click here to register)
Advocacy
Do it For GM1 Runs August 1-31!
Every year, we invite GM1 families and friends to join us for our annual Do It For GM1 campaign. The concept is easy: pick something you love (running, cooking, gaming) and share it with your friends and family to raise awareness and funds for GM1. This year’s efforts are geared towards increasing funding for Enzyme Replacement Therapy so we can continue to develop the treatment for potential use in GM1 patients. This is a critical time for our community. We must raise $600,000 by January 2027 to keep the project moving forward. We know we can “Do It!” Will you join us?
Click here to join our Do It For GM1 fundraising team.
Click here to order Do It For GM1 gear.
Click here for more ideas on getting started.
Calling All Artists! Rare Artist 2026 Open for Submissions
The Rare Artist program, organized by the EveryLife Foundation for Rare Diseases, provides a national platform for artists to advocate through music, poetry, and visual artwork. This year, a new category, short-form video (5 minutes or less), has been added. Your creative passion could help others better understand the rare disease journey. Apply now through July 20.
Click here to learn more.
research & biotech news
Calgary Clinic Opens Phase 3 Nizubaglustat Trial
The M.A.G.I.C. Clinic in Calgary is now recruiting for Azafaros’ Phase 3 clinical trial studying oral nizubaglustat as treatment for individuals living with late-infantile and juvenile forms of Niemann-Pick type C disease and late-infantile and juvenile-onset forms of GM1 gangliosidosis or GM2 gangliosidosis. Enrollment is currently active.
For a full list of locations recruiting for this study, click here.
For full participation details, click here.
Cure GM1 Attends BIO International
The American Society of Gene & Cell Therapy held its annual meeting May 11-15, and numerous stuCure GM1 Attends BIO International
Cure GM1 co-founder Christine Waggoner was on-hand at the 2026 BIO International convention in San Diego to connect with others in the rare disease space of the biotech industry. Progress in biotech takes more than science alone. It takes advocates who raise awareness, build partnerships, and keep patient needs at the heart of every conversation. Every connection we make is a step forward for GM1, and we were proud to represent the GM1 community at this event.
Azafaros Publishes Nizubaglustat Update
Azafaros recently published its progress on utilizing nizubaglustat as treatment for lysosomal storage disorders including GM2 and Niemann-Pick Type C disease in the “Molecular Genetics & Metabolism.” According to the study, nizubaglustat demonstrated “a favorable safety profile and potential clinical benefits by reducing disease progression and seizure burden in participants with GM2 gangliosidoses or NPC, supporting advancement to a phase 3 trial in a larger cohort.”
Read the full update here.
Prenatal Gene Therapy clinical Trial Announced
In May, pediatric surgeon Tippi MacKenzie (University of California, San Francisco) shared the design of a phase 1, first-in-human trial of a new prenatal gene therapy for the treatment of GM1. Dr. MacKenzie provided the update as part of a larger presentation titled “In Utero Therapeutics: Engineering Fetal Futures with Cell and Gene Therapy” at the 2026 American Society of Gene and Cell Therapy annual meeting. Researchers are hoping to enroll five participants.
To learn more, click here.
View our complete GM1 Clinical Trials Guide.
download Our Miglustat Guide for GM1 Families
Overwhelmed by conflicting information on miglustat available online? Cure GM1 has prepared an honest, plain-language summary of research surrounding miglustat for use with GM1, including studies that raised hopes, those that raised concerns, and where the science currently stands.
Click here to view it now.
Fundraisers
Register Now for Our Do It for GM1 Team!
August is just around the corner. Sign up now and start planning what YOU will do for GM1!
Double Your Impact!
If you’ve ever wanted to support Cure GM1 while making a significant splash, now is your time. We are actively seeking a matching gift partner to kick off this year’s campaign. If you are interested in doubling your own impact for the GM1 community, email jess@curegm1.org.
Sponsors Needed!
Our International GM1 Community Conference is Oct. 2, and we’re still in need of sponsors to join our team. Email christine@curegm1.org for more details.
In Memory
Please Join Us in Sending Love To Our Grieving Families
June was an especially hard month for our small community. We lost four beautiful lives far too soon: Aksinya, João Miguel, Armand and Ada. We continue to hold space for every family impacted by GM1 and recommit to forging a path toward making treatment a reality. Please join us in holding space for these families.
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