Research for Duchenne Muscular Dystrophy

When Harrison was four years old, he was diagnosed with Duchenne Muscular Dystrophy (DMD), which causes muscle weakness. Now aged nine, he is participating in one of the clinical trials at the Somers Clinical Research Facility (CRF) at Great Ormond Street Hospital (GOSH). Here, Harrison’s father, Alex, shares their experience of taking part in research.

Living with DMD

"Harrison has been on steroids since he was diagnosed and he’s not the little boy we remember at four years old. The steroids make him quick to feel emotion and sadness. It’s a very debilitating condition and he gets tired doing normal things. Harrison finds it frustrating that he can’t keep up with his brother, William, and his friends. But he always throws himself into activities and puts on a brave smile."

Making the decision

GOSH patient with his parents
"My wife and I talked to lots of families who have been through clinical trials and who have children with this condition. We had to consider some unknowns about the treatment and that it might be stressful for Harrison. We also know that there’s a one-in-three chance that he might be on the placebo. If it’s the case that he’s on the placebo, we might be in the position where we see nothing changing for Harrison, while the other children do well. However, we know that it is currently the best way forward.

"Harrison is a strong-willed little lad and very stubborn. We talked about his muscles and said that this might help him become stronger. We also said that it might help him walk and play with William. He was very excited that it might mean he can jump. We got him as much information as we could and talked about blood tests because he doesn’t like them.

"Before the trial started, Harrison was asked to give his assent. Together with my wife and Professor Francesco Muntoni – who is Director of the Dubowitz Neuromuscular Centre at GOSH – I went through the trial carefully with Harrison, but that first time he said ‘no’. He still feels a little that it is ‘daddy’s idea’ but agrees it’s a good decision. He increasingly understands the importance of the trial, and that he’s doing it for himself and to help other children."

The Research Nursing team in the CRF

GOSH patient with a research nurse
"Starting the trial can be a tense and worrying time for the family but the team in the research facility have been fantastic. The Research Nurses appreciate that we are in a challenging situation and really support us. They also make Harrison feel like the centre of attention, without worrying him. No child likes to have blood tests but Harrison gets to play the Xbox and go out for dinner, and so there are things about the day he likes.

"I feel blessed that we have one of the best hospitals in the world on our doorstep and that Harrison gets the chance to be part of a clinical trial. Our greatest hope is that through research and clinical trials, better treatments will be found for DMD."