Eliza is a wonderfully bright and spirited little girl. Her smile fills the hearts of her parents and big brother, helping them find joy even in difficult times. Eliza was diagnosed with Sanflippo Syndrome (MPS III A) when she was three and a half years old. Since that time, so many of the skills she once had – including her speech – have faded away. Amazingly, she was the first patient in an experimental gene therapy trial in May 2016. There is hope that this treatment will stop further progression of the disease. As a family, they continue to look forward with love and hope for their lives together.