Blog

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.