Vision

A cure for Lafora disease.

Goals

1. 1.Get the ION283 Safety Study fully-funded and support future development efforts of the ION283 asset. We must fund the remaining clinical costs ($150k), so all 10 patients can complete testing the potential treatment, then support efforts to make it widely available for our patient community.
2. 2.Establish Clinical Centers of Excellence and Guidelines for clinicians to care for Lafora patients.
3. 3.Expand resources to support and connect Lafora families worldwide. We seek to grow caregiver education and resources and improve platforms for families to connect, communicate, and engage with our cause.