Sims Family

04.24.2026

When Charlie was 6, he was diagnosed with CLN1 Batten disease. His parents were told he would die. They raced against all odds to get him to a genetic treatment never used before. Amazingly, they won. 

Together with the help of their son's doctor, they successfully obtained access to a deprioritized gene therapy that had been sitting unused on the shelf of a biotech company. They got the medicine to Charlie, and he paved the way for 6 other children in desperate need to access the same medicine.

They had so much hope to stop the disease and save him… but for Charlie, the medicine came too late. Today, he is 9 and his disease is relentlessly progressing. They have accepted that the treatment won't save Charlie, but at least they can tell themselves they tried, and for that effort, Charlie might yet still save others. 

That chance to try is something millions of others like them will never have the opportunity to say.