'It's cruel that I cannot get muscular dystrophy drug'

A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available in Northern Ireland.

Alfie, from Newry, County Down, was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years old.

His dad Jamie Pentony said the new drug, Givinostat, could slow his son's incurable condition down, but the Belfast Trust did "not have the capacity" to offer it.

The Belfast Trust said it was not in a position to proceed with the implementation of Givinostat as "additional staffing resources would be required to ensure the treatment could be provided safely".

There are many types of muscular dystrophy.

They are inherited genetic conditions that gradually cause the muscles to weaken, leading to an increasing level of disability.

DMD is one of the most common and severe forms - it usually affects boys in early childhood and many people with the condition will only live into their 20s or 30s.

Speaking on BBC Radio Ulster's Nolan Show, Mr Pentony said there were 13 boys in Northern Ireland who could benefit from the drug.

"Who is going to tell the 13 kids that could avail of this that some of them might not be walking next year?

"Time is muscle when it comes to Duchenne. Your lung is a muscle, your heart is a muscle and most of the boys die from heart failure.

"They [the Belfast Trust] are telling us they do not have the capacity, they do not have the nurses to take the tests, that they do not have the neurologists to look after the boys whilst on this drug."

Alfie said his condition could stop him from walking, but doctors have told him that because he was "doing so well, it will take a while for that to happen".

"The drug would really help me, it would help me a lot, but there are other children that will stop walking before me, so it's important for them to get it as well," he said.

"It's cruel they are not giving this drug to kids in Northern Ireland with this disability. We all have the right to have something that could help."

The anti-inflammatory drug, Givinostat, has been approved for use in the UK by the medicines regulator and is available early for people who meet strict criteria, on the NHS.

Individual trusts must apply to take part in the Early Access Programme (EAP).

The Belfast Trust said: "The drug is available for an Early Access Programme (EAP) for eligible patients, pending decisions about wider access following the National Institute for Health and Care Excellence (NICE) recommendations.

"Currently, Belfast Trust is not in a position to proceed under the EAP. We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them."

Speaking in the assembly at Stormont on Tuesday, Health Minister Mike Nesbitt said that several politicians had approached him to "ask about the drug".

"On the face of it, and I'm not a clinician, it looks like it is a revolutionary and very positive development," he said.

"I would be keen to see it rolled out at pace.

"I am following up with the Belfast Trust to further understand their decision-making process."

Una Farrell, from the charity Duchenne UK, said: "It's just hard to understand with a disease that is so cruel and progressive, and time is muscle, why children cannot be given access to it now.

"We understand that resources are a huge issue, but it's been known for several years that this drug was on its way.

"There is no cure for muscular dystrophy - finally there is a treatment that the data suggests can slow down the progression."

Routine health service access to Givinostat will depend on guidance from health assessment body NICE.

Northern Ireland's Department of Health said it fully understood that patients and families want to access the best available health service treatments and drugs.

"The department maintains a formal link with NICE. In practice, this means that treatments that have been recommended by NICE for routine use in the NHS in England are also routinely available in Northern Ireland.

"NICE are developing guidance on the clinical and cost effectiveness of Givinostat for the treatment of DMD in people six years and over, with a committee meeting scheduled for May 2025."

You can listen to the full Nolan Show interview on BBC Sounds here.