What Is GM1 Gangliosidosis?
GM1 Gangliosidosis is a rare, inherited genetic disorder that affects the brain and nervous system by preventing the body from breaking down certain fats, leading to progressive neurological damage.
If you’ve never heard of GM1 Gangliosidosis before, you’re not alone. Even many medical professionals may only encounter it once or twice in their careers. For families facing this diagnosis, it can feel like being dropped into a foreign country without a map. This guide is here to help you understand GM1 Gangliosidosis in clear, human terms without overwhelming medical jargon.
We are honored to share that our very own “Sweet Iris” and the mission of the Cure GM1 Foundation were recently featured in a moving segment on KRON 4 News. As we observe Rare Disease Day, this coverage shines a vital spotlight on the realities of living with GM1 Gangliosidosis and the urgent need for continued research and funding.
Watch the KRON 4 News Feature:
A Journey of Resilience and Advocacy
The segment follows the journey of Iris Dooley, who was diagnosed with juvenile GM1 Gangliosidosis in August of 2013 [00:00]. While doctors initially gave a 10-year life expectancy, Iris recently celebrated her 18th birthday—a milestone that speaks to her incredible spirit and the dedication of her parents, Doug Douly and Christine Waggoner [01:43].
GM1 is a “truly brutal” degenerative neurological disease that destroys the brain and spinal cord [00:18]. As the KRON 4 News report highlights, the disease has robbed Iris of her ability to walk, talk, and eat, yet her smile remains a beacon of hope for her family and the entire rare disease community [01:36].
Turning Pain into Purpose
Since Iris’s diagnosis, the Dooley family has turned their personal struggle into a global movement. Through the Cure GM1 Foundation, they have:
- Raised millions of dollars to fund critical GM1 research [02:04].
- Help draw attention to GM1 which lead to five human clinical trials [02:04].
- Advocated for Newborn Screening, which Dr. Tippi MacKenzie of UCSF notes is essential for treating the disease before irreversible damage occurs [04:02].
Though Iris herself was eventually unable to qualify for the trials her parents fought so hard to start due to the progression of her disease, the family continues to push forward [02:25]. As Doug Dooley shared with KRON 4 News, “You just want to solve it for others because you don’t want anyone going through this” [02:40].
How You Can Help This Rare Disease Day
Rare diseases like GM1 affect fewer than 5,000 people in the U.S., but as Christine Waggoner reminds us, “rare diseases actually aren’t that rare” when you consider the millions of families globally facing similar battles [04:38].
We need your help to ensure that life-saving treatments don’t sit “on the shelf” while children are suffering [04:17]. You can make a difference today by:
- Sharing this story to increase awareness of GM1.
- Volunteering your time to support our mission.
- Donating to fund the next breakthrough in research.
Donate Now to Support a Cure
Every contribution brings us one step closer to a world where no child has to face the devastating effects of GM1 gangliosidosis. Together, we can provide the “fighting chance” these children deserve [04:53].
For more information on our initiatives and how you can get involved, visit CureGM1.org.
FAQs
What is GM1 Gangliosidosis?
GM1 Gangliosidosis is a rare, inherited neurological disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. It is caused by a genetic mutation that prevents the body from producing enough of the beta-galactosidase enzyme, leading to a toxic buildup of GM1 ganglioside in the body.
How rare is GM1?
According to the National Institutes of Health (NIH) occurring in approximately 1 in every 100,000 to 200,000 newborns. Because it is so rare, funding for research often depends on private donations and foundations like Cure GM1.
Is there a cure for GM1 Gangliosidosis?
Currently, there is no FDA-approved cure for GM1. However, the Cure GM1 Foundation has successfully attracted interest and collaboration that resulted in five human clinical trials to date. Currently, however, there are only two active clinical trials which are currently enrolling as of early 2026. Research is ongoing to find treatments that can be administered as early as possible to prevent irreversible damage.
How can I support the mission of Cure GM1?
You can support our mission by donating directly to fund medical research, volunteering your time, or sharing our story and the KRON 4 News segment to raise awareness on social media. Awareness is the first step toward a cure.