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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.