Blog

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.