Drug Offers Hope for Boys with Duchenne Muscular Dystrophy
Families Fight for Access to Life-Changing Treatment
A promising medication offering a potential slowdown in the progression of Duchenne muscular dystrophy is currently available at no cost, yet access remains uneven across the United Kingdom, sparking a desperate struggle for families facing a heartbreaking postcode lottery.
A Race Against Time
Ten-year-old twins, Jasper and Arabella, are battling Duchenne, a genetic disorder that progressively weakens muscles. While Arabella is a carrier, Jasper’s condition is more severe. The drug, givinostat, has demonstrated the ability to slow the disease’s course and is being provided free of charge by ITF Pharma while awaiting approval from the National Health Service (NHS).
Despite being part of an early access scheme since November, only a limited number of NHS trusts are distributing the medication, with even fewer in England. According to the Centers for Disease Control and Prevention, approximately 1 in 3,500 to 5,000 male births are affected by Duchenne muscular dystrophy. (CDC – Duchenne Muscular Dystrophy)
“We try and live in the moment and, in this moment, there’s an opportunity to make a massive change to Jasper.”
—Rosie Day, Mother of Jasper
Cost Concerns and NHS Delays
Charity Duchenne UK has emphasized the urgency of the situation, noting that families are facing a critical window to benefit from givinostat. Some NHS trusts are delaying access or establishing priority lists, citing the annual monitoring cost of approximately £1,900 per patient as a barrier. Parents have directly appealed to Health Secretary Wes Streeting for intervention, but the government maintains that access decisions rest with individual NHS trusts.
Emily Reuben and Alex Johnson, founders of Duchenne UK, stated: “As time ticks by, more boys are losing out on their chance to access givinostat. This is a simple treatment, which can be easily managed at home, and requires uncomplicated blood tests to monitor. It’s free to the NHS and could offer real hope for patients and their families. The delays are cruel and the postcode lottery is unjustifiable. We are calling on the NHS to urgently make this available to both ambulant (walking) and non-ambulant boys.”
Currently, all health boards in Scotland, along with those in Swansea and Cardiff, are rolling out the drug. Leicester Royal Infirmary has become the first hospital trust in England to administer the medication. Other trusts, including those in Manchester, Liverpool, and Newcastle, and the Evelina, have yet to follow suit, though Great Ormond Street Hospital is preparing to offer the treatment.
An Evelina spokesperson explained that they are contacting eligible patients and prioritizing those at the highest risk of losing mobility. The NHS anticipates a National Institute for Health and Care Excellence (NICE) review in July 2025, with potential for expedited access if ITF Pharma offers a cost-effective price.
The NHS representative added that trusts are being guided on early access schemes, acknowledging the need for additional resources to administer new treatments. For families like the Days, every day without access represents a potential loss of function for Jasper, highlighting the critical need for swift action.
“It’s a lottery, basically, depending on where you are in the country … and within NHS trusts themselves. Every day that passes by is a day that we can see Jasper decline and see him struggling with his muscles.”
—Rosie Day, Mother of Jasper