Alt text ION283 Therapy Roadmap Dr. Minassian's team recently submitted an Investigational New Drug application to the FDA for a safety study of ION283 ASO therapy. If approved, the safety study will enroll around 10 patients and will be held at UT Southwestern Medical Center in Dallas, Texas. Following FDA review, the information about the safety study will be found on clinicaltrials.gov, and Chelsea's Hope will send out further communication. Step 1: Drug development - lonis developed ION283, an ASO drug that inhibits Gys1, blocking glycogen build-up. Step 2: Animal Model Studies - ION283 was tested in in vitro cell lines as well as rodent & non-human primate models. The results were promising, showing halted accumulation of Lafora bodies and no adverse effects. We are here! Between steps 2 and 3. Step 3: Safety Study (2 years) - This is a will be the first trial of ION283 in humans, to determine the safety and ideal dosage level. Step 4: Efficacy Studies - A pharmaceutical company will need to purchase the drug and conduct phase 2/3 studies, which will enroll a larger number of patients with Lafora Disease to further understand the effectiveness of the drug. Finally, that leads us to FDA APPROVAL OF ION283 FOR TREATMENT OF LAFORA DISEASE.