ROME Daniels (UK)

Rome is 2 years old. When he was born he presented with a lactic acid level of 12 and spent a month in intensive care. Rome was diagnosed deaf after failing his newborn hearing test and was fitted with hearing aids shortly after. Rome was diagnosed with ECHS1 at age of 9 months after undergoing a skin and muscle biopsy.

Rome was learning well, was a couple months behind for his age range but was saying dad mum, nearly sitting unaided and rolling over, until November 2018 when he caught RSV and was left brain damaged. He lost all skills, smiling, body control and lost his sight. After spending 2 months in hospital, Rome is back at home doing very well, he had a feeding tube fitted but hasn’t regained much skills since.

We’re hoping that gene therapy will give our kids a chance in living a happier, healthier life, giving them the chance to explore the world without this horrible disease.