Do you know about Lafora Disease? This rare genetic disease begins during adolescence. Your child starts their life healthy and whole, but then they decline, cognitively and physically, until they cannot care for themselves at all. There is currently no form of treatment. The families who have suffered from this have banded together and formed Chelsea’s Hope Foundation named for the beloved Chelsea, who, like all those diagnosed to date, lost her life to Lafora disease.

“Symptoms include epilepsy, ataxia, childhood dementia, cognitive decline, and difficulty speaking, walking, and eating. There is currently no cure; the disease is always fatal.”

Families facing Lafora have started a clinical trial at UT Southwestern, with an enrollment goal of 10 patients. All of the costs of this trial are being funded by patient families, further highlighting their inspiring resolve.

Dr. Berge Minassian shared: “I am pleased to report that our Safety Study officially began last December. So far, we have enrolled four patients who have already received their first dose of ION283. Patients five and six are making arrangements to be enrolled this month and schedule their first treatment. We will continue to enroll patients as enough funding becomes available to begin their treatment.” (UPDATE: Patients five and six have been enrolled.)

Watch this video to learn more about the urgent need to enroll the final 4 patients.

If your employer, organization, or foundation would like to make a contribution, or if you are interested in learning more, please visit Chelsea’s Hope, or feel free to contact them directly via email – [email protected]