Now Charlie can join in with the other children

JENNIFER HANDT realised within a year of her son Charlie’s birth, as he began missing crucial milestones, that he was not like other children.

The 47-year-old copywriter from Darien, Connecticut, says: ‘He struggled to turn himself over and still wasn’t walking. I thought it was strange but didn’t think it would be anything too serious, so we took him to see a physical therapist.’

But Charlie did not improve and, at three, he was referred to a neurologist who diagnosed him with Duchenne muscular dystrophy (DMD) – a genetic condition that affects only males and which was once a death sentence.

Jennifer adds: ‘It was devastating because I knew what this disease did and how it could affect Charlie’s life, but I was also hopeful. I’d read about the amazing experiments which were going on with gene therapies, and decided early on that we weren’t going to let this diagnosis ruin our lives – we were going to find a cure for Charlie.’

Jennifer and her husband Rick were able to get Charlie on to the Elevidys trial, and in 2022, aged three, he was given an infusion.

She says the improvement in Charlie’s mobility has been striking.

‘He’s begun to show some very promising signs,’ she explains. ‘Charlie is able to get up off the floor without using his hands or another part of his body for leverage – the hallmark test for how advanced DMD is. This isn’t something he could do before.

‘He can climb up stairs easier than before and takes part in PE lessons for short periods.

‘He is able to act like a normal, healthy child – and it is amazing.

‘We know this drug isn’t a cure, but it could buy Charlie enough time for a cure to be found in the future.

‘This drug should be available to every child who needs it.’