The drug givinostat - used for Duchenne Muscular Dystrophy management - remains unavailable to boys and young men who can no longer walk and this had plunged Alex Clarke's family into uncertainty15:53, 01 Jun 2026A 12-year-old boy with a rare muscle-wasting condition has finally started a life-changing NHS treatment after years of uncertainty for his family.‌Ben Clarke has Duchenne Muscular Dystrophy (DMD), a severe, progressive genetic disorder that mainly affects boys and causes muscles to weaken and break down over time. He is among hundreds of patients set to benefit from givinostat after the drug was approved for use through an early access programme in certain NHS Trusts.‌But his father Alex Clarke said the fight is far from over, warning other families could lose sons before being given the same opportunity. Recalling the moment Ben found out the drug had been approved, Alex told the Mirror: "The smile of 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."‌Givinostat is designed to slow the progression of the condition and help preserve muscle function for longer. However, it has only been approved for boys able to walk.Although this is a happy moment for the family, Alex said the campaign is not over, as givinostat has only been approved for ambulant boys.‌Ben was four years old when he was diagnosed with DMD in 2017. His parents initially sought medical advice after noticing concerning symptoms, and after a doctor suspected Duchenne, the family turned to the internet to research the condition.Alex, from Sonning Common, Oxfordshire, told the Mirror: "At the time, the guidance online was very wrong and said that he wouldn’t live beyond his 12th birthday. Which at the time, was heartbreaking - we were heartbroken. You have this anticipatory grief, this feeling of what does the future hold and what does it look like?"Ben has since turned 12 and recently began taking the treatment his family spent years fighting to access. Alex said the family made a decision just weeks after the diagnosis to focus on giving Ben the best life.‌“We have an opportunity here to give Ben his best life. We don’t know how long it’s going to be. We don’t know what impact it’s going to have, but we have to make sure that we do have is amazing."Now 12, Ben attends mainstream school full-time and is described by his dad as “the sunshine that lights up a room when he walks in.”‌While Ben can still walk, he relies on a wheelchair for longer distances and his family have watched as everyday tasks become increasingly difficult.“As a parent, that is really hard to see and really hard to experience, you’re there to protect your child and there to fix stuff when stuff is broken or wrong, and you can’t - there’s nothing you can do except be there to love and support him.”The family spent more than a year campaigning for access to givinostat alongside Duchenne UK through its ‘Time is Muscle’ campaign, after Ben’s trust did not take on the early access programme.‌“It was really hard to go from 24 trusts saying no to 21 trusts saying yes and 3 trusts saying no, and one of those trusts being where Ben sits and so we couldn’t access it under the Early Access Program.”Duchenne UK’s co-founder and Chief Executive said: “It has taken NICE (National Institute for Health and Care Excellence) 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 and they lost mobility and function, which once lost, can never be regained in DMD.‌“Meanwhile, under this guidance, patients who are unable to walk or stand, will still be unable to access givinostat. This experience has underlined the need for urgent reform to ensure that drugs for rare, severe, paediatric-onset diseases like DMD, are being assessed and valued appropriately and to prevent unnecessary delays for children and families seeking access to them, ensuring that they can benefit from the scientific advances being made.”Alex said families with non-ambulant sons are still facing the heartbreak of knowing a treatment exists but remains out of reach.‌He explained: “We have to keep in mind that there is a huge part of our community who are non-ambulant and are in wheelchairs are unable to access this, and we just hope that we can really get that over the line with the the next round of this campaign.”“The regulatory and approval process that we have create this postcode lottery whereby if you happen to live in the wrong place at the wrong time, you can be left behind and that is absolutely unfair.”“The longer we delay getting therapies and treatments to our boys, the less time they have and I think that’s what’s really important now with these non-ambulant boys in our community and young men, is they deserve the chance that all of these ambulant boys have got.”Article continues below“It’s not their fault and it’s really unfair that time is not on their side, that their muscle is being impacted by, by not having access to a therapy and a treatment that is proven to be safe and effective.”