A 12-year-old boy with Duchenne Muscular Dystrophy (DMD) has started a life-changing NHS treatment after years of uncertainty, but his father warns other families may miss out. Ben Clarke, from Sonning Common, Oxfordshire, began taking givinostat through an early access programme after his family campaigned for over a year.
Ben’s father, Alex Clarke, said the fight is not over, as the drug is only approved for boys who can still walk. He told the Mirror: “The smile on his face was incredible. He is just so happy that it was available and he could now get this drug. There’s been no negative side effects to this point and he says he’s feeling really good on it.”
Ben was diagnosed with DMD at age four in 2017. His parents initially sought medical advice after noticing symptoms, and a doctor suspected Duchenne. Alex recalled the devastating online guidance at the time: “It said that he wouldn’t live beyond his 12th birthday. Which at the time, was heartbreaking.” Ben has since turned 12 and attends mainstream school full-time, described by his dad as “the sunshine that lights up a room.”
Givinostat is designed to slow progression and preserve muscle function, but has only been approved for ambulant patients. Duchenne UK’s co-founder and Chief Executive criticised the lengthy NICE process, saying: “It has taken NICE almost two years to make a decision. During that time, many families were left without access to this medicine, while their child’s condition progressed.”
Alex stressed that non-ambulant boys remain excluded: “We have to keep in mind that there is a huge part of our community who are non-ambulant and are in wheelchairs unable to access this. We just hope that we can really get that over the line with the next round of this campaign.”
