Safety Study Update – February 2025

We are excited to share an update about the ION283 Safety Study: the team at UTSW can continue enrolling patients. 

In December 2024, the first patient in the Lafora Disease Safety Study at the University of Texas Southwestern Medical Center (USTW) received their first dose of ION283, a treatment designed to halt disease progression. Last month, that patient completed their one-month check-up with no safety issues detected. This allowed a second patient to receive their first dose of ION283 in January. Earlier in February, the second patient completed their one-month safety check. The safety board at the university reviewed the safety data, giving an all clear to proceed as planned with the study.

The first ten families who applied to participate in the study will receive notification from the team at UTSW when there is enough money available to enroll them in the study. We are pleased to share that the third patient is en route to Dallas, and the fourth has been notified.

As a community, we must continue raising funds so all 10 patients can receive treatment. The data collected from this study will help accelerate the approval process so that we can expand access for other patients to receive this drug. However, we need to complete the enrollment for this study first and get the data we need. So far, we have raised more than $977,000 as a community. We must reach 1.5 million dollars by May 2025 to fully fund the study. 

We will continue to keep the Lafora community informed about the progression of the Safety Study. Any questions not answered in our FAQs about the study can be directed to Chelsea’s Hope at info@chelseashope.org.