As the year comes to an end, we remain focused on creating connection by keeping our rare disease community informed of advocacy opportunities, research news and community updates. Join us in learning more about our efforts to push for a cure for GM1 gangliosidosis.
This month as we give thanks, we want to express our deepest gratitude to this strong and resilient community. We’re thankful for all the truly kind support and generosity that fuels the work we do. While we truly wish that no one had to suffer from GM1 gangliosidosis.
Let’s take a minute to celebrate Christine, Connie, Niclas, and Kevin for “Doing It for GM1.” Their combined efforts have raised over $35K! The month closed with great news from SylamoreBio, who received a $3 million NIH grant to advance their innovative GM1 research.
What an incredible month August was! Our 2025 International GM1 Community Conference brought together participants from around the world. We were excited to hear from Azafaros that the first trial participant was enrolled in Portugal. We witnessed the power of our shared hope, courage, and commitment.
Our annual conference is just three days away on August 8th, and we can’t wait to connect with families from around the world. We’re thrilled to offer live translation services this year, ensuring our global community can fully participate.
This month, our hearts are heavy as we said goodbye to another precious child, sweet Daxton, who passed away just before his fourth birthday. The heartbreaking reality is that most children with GM1 won’t see their fifth birthday—an unimaginable burden that no family should ever have to carry. We cannot and will not remain invisible in the face of this devastating disease.
This month brought exciting advances with the announcement of the first successful personalized CRISPR gene editing treatment for a baby with a rare disease. Many have asked about the implications for GM1. This personalized treatment developed for CPS1 deficiency addresses a different condition than GM1.
As May begins, we’re filled with anticipation for GM1 Awareness Day on May 23rd. This is a month-long effort to shine a light on GM1 gangliosidosis and the remarkable community that surrounds it. We’ve submitted 12 proclamation requests to officials across the country.
Ten years ago this month, on April 17, 2015, the U.S. government approved the Cure GM1 Foundation to be the first and only 501(c)(3) entirely dedicated to GM1 gangliosidosis research, drug development and awareness. Over the course of a decade, our efforts have grown significantly. What began as a more personal search for hope became a global movement that has transformed the landscape for this rare disease.