Cure GM1 Catalyst
Happy May!
This is our favorite month because it means International GM1 Gangliosidosis Awareness Day 2026 is almost here!
For us, May 23 isn’t just a date on the calendar. It’s a powerful opportunity to honor the journeys of those impacted by GM1 and shine a powerful light on its impact on our global community.
Already, we’ve seen GM1 parents step up in inspiring ways: requesting official GM1 Day proclamations in their states, sparking conversations with local leaders, and bringing visibility to a disease that deserves far more attention. From social media campaigns to community events, awareness is spreading across borders as supporters around the world join in.
What makes GM1 Awareness Day so special is the energy behind it. It’s the sense that every voice matters and every action counts. We can all do something! Invite a friend to learn about GM1. Reach out to your local officials. Share why this day matters to you. YOUR VOICE is what makes GM1 Day so powerful.
Together, we can turn awareness into action and bring us one step closer to better treatments, stronger support, and ultimately one day, a cure.
Let’s make GM1 Awareness Day 2026 unforgettable! If you’re looking for more ways to get involved, message us at info@curegm1.org.
Community Engagement
Remembering Batul
““My daughter, Batul, did not deserve this. At 2 years 7 months, she had seen more hospitalizations than most adults. Every time she came home, she had won a battle with GM1. But each battle cost her through physical toll and losing abilities.” — Batul’s father
2026 (Virtual) Conference Registration is Open!
Registration for the Oct. 2 event is FREE for GM1 Families. If you are longing for connection, information and support, this is the place for you. This virtual event makes it easy to attend, no matter where in the world you’re living with GM1. We hope to see you there.
Click here to visit the conference website (agenda still in the works).
Mail info@curegm1.org for a sponsorship package.
Don’t Miss the Next GM1 Community Chat: May 21, 2 p.m. PT / 5 p.m. ET
This is your chance to connect with other GM1 families, ask questions, and find honest feedback and support. Registration is required to ensure the safety of our members.
Advocacy
Help Push for Newborn Screening of GM1 in California
Cure GM1 board member David Law has put together a Change.org campaign to push for newborn screening of GM1 in California. Together, we’re calling on the California Department of Public Health and other state leaders to fund and implement a pilot screening program to assess feasibility and impact. The effort could identify GM1 before it irreparably advances in young babies. It could also allow us to gather even more data about GM1 that could one day lead to treatment and a cure.
Click here to sign the petition now.
Click here to learn about our other newborn screening efforts.
Rare Revolution Magazine Focuses on Lysosomal Storage Disorders
This issue of Rare Revolution Magazine includes information on breakthroughs being made in the lysosomal storage disorder area. Topics include the evolution of LSD classification; one father’s journey seeking answers for his son; a case for newborn screening for Niemann-Pick and other LSDs; and a new “Raregivers” partnership with Crisis Text Line.
upcoming Events of Interest
May 23: International GM1 Gangliosidosis Awareness Day! Share your story with friends, family and local community media to raise awareness of GM1. Tag us @curegm1 so we can share your stories, and don’t forget to download our free profile photos, coloring pages and more!
June 4-7: International Symposium on MPS and Related Lysosomal Diseases. Florence, Italy. Includes family and children’s programming.
Sept. 15-16: C-Path Global Impact Conference 2026. Washington, D.C. For regulators, industry leaders, scientists, clinicians, those with lived experience, and advocates.
research & biotech news
Online Focus Group for Caregivers of a Child with GM1 Gangliosidosis (Type 2a and 2b)
The Cure GM1 Foundation is supporting recruitment for a global research study conducted by Sprout Health Solutions on behalf of the non-profit organization, Critical Path Institute. The study is funded by the FDA.
By participating in this study, you will:
Help researchers better understand the lived experience of individuals with GM1 gangliosidosis and their families by providing feedback on two visuals describing the disease journey of GM1 types 2a or 2b
This study is for:
Primary caregivers of children living with GM1 type 2a (late-infantile) or type 2b (juvenile), and Bereaved primary caregivers whose child with GM1 (type 2a or 2b) passed away within the past 12 months
If you are eligible, you may be invited to take part in:
An online focus group (about 90 minutes)
Participants will receive $150 USD (or the equivalent value in local currency) for participating in the focus group, plus $25 USD (or local equivalent) for submitting confirmation of diagnosis.
To learn more, please contact the study team at GM1-study@sprout-hs.com.
Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 43% funded by the FDA/HHS, totaling $20,724,703, and 57% funded by non-government source(s), totaling $27,346,613. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
The Critical Path for Lysosomal Disorders Consortium is funded by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) through grant U18FD005320. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.
Click here to download a flyer about the focus group.
Mayo Rochester Added to List of Active Recruiting Sites for Azafaros Trial
The study is evaluating the safety and efficacy of nizubaglustat for individuals diagnosed with GM1 and GM2 gangliosidosis, including Tay-Sachs and Sandhoff disease.
- Primary Eligibility: 4 years and older and SARA score between 3 and 30
- Location: Mayo Clinic Rochester, MN, campus
- Contact: Brad Jorde (507) 293-3609, jorde.bradley@mayo.edu
For a full list of participating locations worldwide, click here.
Critical Path Awards $456,000 to Advance Next-Gen Therapy for Gaucher and GM1
Critical Path Institute announced a $456,000 grant to James Shayman, M.D., at the University of Michigan, to support development of a brain-penetrant therapy for glycosphingolipid storage disorders. The funded project focuses on developing a therapy to address neurological symptoms that remain untreated by current options.
Click here to read the full press release.
Fundraisers
Help us Make ERT a Reality
Our main focus this GM1 Day is to raise funds toward Enzyme Replacement Therapy. Every donation, no matter the size, helps!
Click below to download / share our ERT brochure.
Rare Moms Club
Mother’s Day is around the corner. Check out our selection of Rare Moms Club gear, available through May only.
Order Your Personalized GM1 Day T-shirts!
Grab your GM1 Day merch now to ensure you receive it by May 23. For a limited time, we are offering personalized options! Email jess@curegm1.org for a link to order a GM1 Day shirt with your child’s name on the back. (You must email us first to set up the link, rather than ordering before you message us.)
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