by Parents whose sons lose the ability to run have described the “nightmare” glow, whom they are faced with the NHS to access a free drug.
The drug givinostat can slow down the progress of the Duchenne muscle dystrophy – keep the children on their feet longer – and is offered free of charge by a drug company, while it goes through the process of the NHS approval.
However, only a few NHS trusts all over the UK and only a few in England indicate the medication, although it has been part of an early access program since November.
According to the charity organization Duchenne UK, families are now facing a “cruel” postcode lottery who gets medicine and is against time.
Some NHS trusts refused to deliver the medication, while others delay it by creating lists that they believe should get it first.
NHS Trusts say that you have to pay for the monitoring of the drug – around £ 1,900 per year per patient – from your own budget.
In desperation, the parents met with the health secretary Wes Streeting to accelerate access.
According to the government, it is due to individual NHS trust to decide whether the medication should be provided.
Duchenne UK supports families in their struggle for givinostat, including Rosie and Pete Day, an investment manager and their 10-year-old son Jasper from Horsham, West Sussex.
The day family has twins Jasper and Arabella at the age of 10 and two older daughters.
Both Jasper and Arabella have the most strongest, with Arabella, a strap of the genetic disorder and jasper because the condition mainly affects boys.
The medication developed by ITF Pharma UK is only given as part of the Early Access program to children who can still run.
Ms. Day said the PA news agency: “We are trying to live at the moment and at this moment there is the opportunity to make Jasper a massive change.
“We won’t heal it, but this drug gives him the best chance of having the early life that he wants and what every parent wants to give his child.
“It’s so close – and yet we’re stuck.
“We are in this process where there is something that could make something that could make a big difference to keep him running, keep him active, keep him in the garden with his siblings, to go up the stairs, all of these things, and it simply feels that we cannot get there quite.
“We are on the finish line to get something that makes a difference, and because of the system we are in, we cannot quite get there.
“The drug will not heal it, but there will be the childhood that you want to give your child – to let your child play football, to get to dog walks, to walk up the stairs, to play the French horn, all the things you dream of doing it.
“We cannot give it because we hold on, even though it is free and there is – we cannot exist.
“It feels like a nightmare because you know that the doctors want to give it, but when it comes to putting the drug into your mouth and seeing what it would do, we are firm.
“It is basically a lottery, depending on where they are in the country … and in NHS it trusts itself.”
Jasper deals under the care of the Evelina London Children’s Hospital, which only now begins to contact patients and creates a list of priority for who receives the medication first.
“Every day that gets past is a day when Jasper decreases and see how he has to deal with his muscles,” said Ms. Day.
“Two weeks ago he asked me:” When do I get the drug, mom? “
“In a situation, we are firmly about who will make these decisions about who gets the drug – whether Jasper gets it or another boy.
“It is heartbreaking for every boy because in the end someone does not get the medication on the basis of the current criteria at this moment.”
She added: “If Jasper stops going, we won’t get it back.
“It is important every day. Six months ago, Jasper was able to easily go up and down the stairs. He can’t do that now and Pete has to push him up the stairs every night.
“Nevertheless, we cannot give him anything that is as simple as two spoons full of medicine and blood test, and a little resources that look at the blood testers – that’s what stands in our way.”
Ms. Day said it was unclear how Evelina will “make a fair decision” because knowing who will lose mobility is not a precise science.
Mr. Day said that the process was “very subjective” because there is no linear decline in the disease and the decisions of NHS Trusts are “completely wrong”.
According to Duchene UK, boys whose condition receives a worse risk, the list will fully fall out of the list while waiting for the medication.
Around 500 boys in Great Britain are entitled to givino, which can be taken at home like Calpol. In the first year, the hospitals followed around eight years after follow-up blood tests, followed by twice a year after.
Children may also need an EKG, although they are already part of the routine throat.
Emily Reuben and Alex Johnson, the founders of Duchenne UK, said: “Over time, more boys passed their chance to gain access to givinostat.
“This is easy treatment that can easily be managed at home and requires uncomplicated blood tests for surveillance. It is free of charge for the NHS and could offer the patient and their families real hope.
“The delays are cruel and the postcode lottery is unjustified. We are calling for the NHS to provide this urgently for outpatient (walking) and non -outpatient boys.”
So far, all health authorities in Scotland have been rolling out the medication alongside those in Swansea and Cardiff.
Leicester Royal Infirmary was the first confidence in England that the drug granted, but Evelina does not yet have it and does not have confidence in Manchester, Liverpool and Newcastle.
The Great Ormond Street Hospital is working on the treatment of the medication.
An Evelina spokesman said: “We have started to contact families of all existing patients in Evelina London, who may be suitable for givinostat and set up appointments in the next few months.
“Our clinical team works through our patient lists and is currently prioritizing children who need treatment most urgently or who have a higher risk of losing movement (ambulation) soon.”
An NHS spokesman said: “The first meeting of the National Institute for Health and Care Excellence (Nice) to take this treatment into account in July 2025, and if the manufacturer ITF Pharma can offer an inexpensive price to recommend its use.
“NHS England has published guidelines on the early access programs led by manufacturers, in which trusts cover considerable costs and have to find additional clinical resources for the management of new treatments. We understand that a number of trusts across the country are preparing to offer givinostat through such a system.”
