Sims Family

04.22.2026

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.

04.23.2026

Life for Charlie and his family has been a roller coaster. He started his childhood happy and healthy, then unexplained problems culminated in a fatal Batten disease diagnosis. Hopes and dreams for their futures came tumbling down. But his parents fought for a treatment that he finally received and their roller coaster amazingly shot up to the sky… only to learn a few years later that the treatment came too late. 

Today, Charlie is blind, has lost most of his words, can no longer walk, is back in diapers, and is getting a feeding tube placed soon. He spends much of his time sitting with his parents listening to the movies he once enjoyed watching. They now celebrate the little things, like the huge effort he makes to lift his arms and throw a ball or a giggle when he remembers something funny that his dementia hasn’t yet stolen from him. But their smiles quickly fade as they watch their boys together, Charlie no longer able to say his big brother’s name. The future they once hoped for, painfully obvious that it will never be.

04.28.2026

Charlie and his family used to go bowling and to the arcade together, but they rarely go any more as a family since it’s too hard for him to participate and the loud noise is just too much.

Instead, they find themselves either dividing the family for activities, or spending their weekends anchored at home on the sofa, a life that’s hardly normal for James. While Charlie’s mom helps feed him, his dad sits in the heavy realization that he no longer has hobbies for himself. He works all day, then immediately switches over to dad and caregiver. 

On the ever dwindling chances to get out of the house as a family and spend time with Charlie’s cousins, that brief moment of normalcy is faced with the painful glimpse of what life as a family could have been.

05.01.2026

Bath time is a special time for Charlie and his dad Matt. A quiet moment together at the end of a long day. But Charlie can’t move independently anymore, so his dad has to carry and lift him often, including in and out of the bath. It all takes a toll on Matt’s body as Charlie is almost 80 pounds now, but Matt can’t bear to complain when he knows how much harder things are for his son. So he works out when he can to maintain the strength he needs to care for Charlie. But back pain and aches are now a regular part of his life. At 41, he feels much older, needing pain injections to take the edge off of the constant back pain. In his mind, there’s no choice but to push through it… because there are only so many more of these small moments of joy he might have left with his son.