Charlie is a joyful 8-year-old who brings light to everyone around him — but every day, he faces the challenges of SLC6A1, a rare genetic disorder that causes seizures, autism, anxiety, OCD, ADHD, behavioral difficulties, and cognitive delays.
There was no cure. But today, there is real and urgent hope.
After years of relentless work, SLC6A1 Connect and a global team of scientists have helped develop a breakthrough gene therapy designed to treat the root cause of SLC6A1. The therapy exists — but we must fund it so more children can access treatment before time runs out.
