A 12-year-old boy suffering from a severe type of muscular dystrophy expressed his frustration over the unavailability of a new drug in Northern Ireland, labeling the situation as “cruel.”

Alfie, hailing from Newry in County Down, was diagnosed with Duchenne muscular dystrophy (DMD) at the tender age of four.

His father, Jamie Pentony, shared that the new medication called Givinostat could potentially slow down the progression of Alfie’s untreatable condition, yet the Belfast Trust stated they lacked the necessary resources to provide it.

According to the Belfast Trust, they cannot move forward with the implementation of Givinostat, as “additional staffing resources would be necessary to ensure safe administration of the treatment.”

There are various forms of muscular dystrophy, which are inherited genetic disorders that progressively weaken muscles, resulting in disability over time.

Duchenne muscular dystrophy is recognized as one of the most prevalent and severe types; it predominantly impacts boys in early childhood, and many affected individuals may only survive into their 20s or 30s.

During a recent segment on BBC Radio Ulster’s Nolan Show, Mr. Pentony noted that there are 13 boys in Northern Ireland who stand to gain from this medication.

“Who will inform these 13 children that some may struggle to walk next year?” he questioned. “In Duchenne, time translates to muscle. Your lungs are muscles, your heart is a muscle, and most boys succumb to heart complications.”

“The Belfast Trust has claimed a lack of capacity, stating they don’t have the nurses for necessary testing or the neurologists needed to oversee the boys while undergoing treatment.”

Alfie mentioned that his condition could eventually prevent him from walking, although doctors have reassured him that since he’s “doing remarkably well, such a change may take some time.”

He stated, “The drug would be incredibly beneficial for me; it would make a significant difference. However, other children may face mobility issues before me, making it essential for them to receive treatment too.”

He added, “It’s unfair that this drug isn’t being provided to children in Northern Ireland with this condition. We all deserve access to potential relief.”

The anti-inflammatory medication, Givinostat, has received approval for use in the UK by medical authorities and is currently available under strict eligibility criteria through the NHS Early Access Programme (EAP).

However, individual medical trusts must submit applications to participate in the EAP.

The Belfast Trust confirmed, “The medication is available through the Early Access Programme for qualifying individuals, though decisions regarding broader accessibility will depend on National Institute for Health and Care Excellence (NICE) guidance.”

They further stated, “Currently, the Belfast Trust cannot proceed with the EAP, and we understand this is disappointing for families. The Trust sincerely apologizes.”

During a recent assembly session at Stormont, Health Minister Mike Nesbitt mentioned that numerous politicians have approached him regarding the medication.

“From what I gather, though I’m not a healthcare professional, it appears to be a groundbreaking and positive advancement,” he noted.

“I am eager to see its prompt rollout.”

“I will be following up with the Belfast Trust to gain further insights into their decision-making process.”

Una Farrell from Duchenne UK remarked, “It’s difficult to comprehend why children are being denied access to a treatment for such a harsh and progressive illness when time is muscle.”

“While we acknowledge that resources pose a significant challenge, it’s been well-known for several years that this treatment was forthcoming.”

“With no cure for muscular dystrophy available, this treatment finally offers a method that can help slow the disease’s progression.”

Accessibility to Givinostat through the regular health service is contingent upon guidance from the health assessment body, NICE.

The Northern Ireland Department of Health expressed understanding of patients’ and families’ desires for the best available treatments and medications.

“The department maintains a formal relationship with NICE, meaning that treatments recommended for routine use by NICE in NHS England are also typically available in Northern Ireland.”

“NICE is currently developing guidance on the clinical and cost efficiency of Givinostat for treating DMD in patients aged six and older, with a committee meeting scheduled for May 2025.”

Listen to the full Nolan Show interview on BBC Sounds here.