Robbie

News Stories

Robbie has never had a normal life. He was born with cystic fibrosis, meaning he always had a continual cough, could not maintain his weight, needed constant physiotherapy to clear mucus from his lungs and was in and out of hospital every few months. His education suffered and his whole family has been affected.

When he was born, his parents were told the condition would limit his life expectancy. But thanks to a brand-new therapy called Trikafta, there is hope that he may live to a healthy, happy old age.

Trikafta helps the body to correct a protein made by the CFTR gene – the gene that has the mutations that cause cystic fibrosis.

The therapy has completely changed Robbie’s life. Now 17, he has more energy, he has stopped coughing and he is enjoying a variety of foods. He has not been in hospital for months and, for the first time they can remember, his parents are making plans for the future.

As more medications become available for children like Robbie, precision medicine will become increasingly important to ensure every patient gets the drugs they need to make them well.