"It's part of Archie's legacy"
By Rebekah O'Reilly
Join Our Boys mum hopes to set precedent for rare disease patients as she advocates for Duchenne drug access
Roscommon mum Paula Naughton is calling for a meeting with Minister for Health Jennifer Carroll MacNeill and the Department of Health regarding licensing and access issues surrounding the drug Givinostat.
The Naughton family, known for the Join Our Boys Trust, have been advocating for improved healthcare access for their sons George and his twin brother Isaac since 2012, when they were diagnosed with Duchenne Muscular Dystrophy (DMD), a progressive neuromuscular disease that destroys muscle tissue and is ultimately fatal.
The family tragically lost their eldest son Archie to the disease in 2022, aged just 16. He would have celebrated his 20th birthday on Wednesday, December 17.
“In 2022, ironically the week that Archie died, an Italian company released data showing that a drug called Givinostat had demonstrated in Phase 3 clinical trials that it could slow muscle damage in children with Duchenne muscular dystrophy,” Paula said.
“When Archie was dying, I was telling him that a drug was coming for the boys. Our fight for Givinostat is part of Archie’s legacy and that of every other child and young man that has not been granted the gift of a treatment.
Givinostat is a prescription medicine that has shown an ability to slow muscle damage in children with Duchenne Muscular Dystrophy. It works by inhibiting HDAC enzymes, which interfere with muscle repair, helping to reduce inflammation and scarring.
The European Medicines Agency (EMA) granted Givinostat a conditional licence in the summer of 2025, a four-year wait which Paula said demonstrates a mismatch between patient need in the rare disease space and the urgency applied by pharmaceutical companies and regulators.
“With Duchenne, every day counts because every day they are losing muscle,” she said. “Four years ago, the boys could feed themselves and use their arms. Now they can’t, and they’re only 15.”
Givinostat has been available in the United States to people living with Duchenne for some time, and in the UK under a conditional licence. However, the EMA licence restricts use in Europe to children aged six and over who can still walk and who are taking steroids.“That means only a subset of children will be eligible once the drug is reimbursed. We are told that steroids are a treatment for Duchenne, but the also do a lot of harm when taken long term,” Paula said.
“In countries where they had their act together, all eligible children are receiving Givinostat free of charge under an early access programme (EAP). Nobody in Ireland is receiving it under an EAP,” Paula said.
It is estimated that approximately 110 children and young men in Ireland are living with Duchenne Muscular Dystrophy. As things stand, Paula estimates that only around 40% of that community would meet the current eligibility criteria for Givinostat.
Another significant barrier, she says, is Ireland’s lack of a national Duchenne patient registry.
At present, Irish patients are listed on a registry operated by Action Duchenne in the UK, which Paula says has become increasingly problematic following Brexit.
“We have never once been contacted about a clinical trial, and I know other families are in the same situation,” she said.
“Without being part of an international clinical trial, children will not be enrolled in an early access programme when a drug becomes available.”Paula also acknowledged the role played by former Minister for Health Leo Varadkar, crediting him with supporting the development of clinical trial infrastructure in Ireland and helping secure funding to advance trial readiness.
“He had a great understanding of the issues we were facing from a medical perspective,” she said. “He did a lot to help the Duchenne community.”
Paula is now calling for a direct meeting with the Minister for Health and Bernard Gloster, Chief Executive of the Health Service Executive (HSE), to discuss the steps needed to establish a national registry and to explore ways of ensuring Irish patients can access clinical trials and treatments such as Givinostat.
“This isn’t just about Duchenne. If we get this right, it creates a model for other rare diseases,” she said. “We owe it to every young person who has died prematurely from this disease to have this conversation. It’s not acceptable not to.”
George's recent complex spinal surgeries
George Naughton recently spent several weeks in Temple Street as he underwent two complex spinal surgeries.
The 15-year-old underwent two complex and high-risk spinal fusion surgeries within a three-week timespan. George and his twin brother Isaac have had a tough year with both brothers facing health issues in recent months.
In October of this year, the family shared George's continued struggle with his spine, and noted that he would be going to Temple Street for surgery. This was followed by a second surgery on Friday, November 21.
The family later shared their relief that surgeons had “worked their magic,” adding that George's surgery went "very well". He then since spent time recovering on St Joseph's ward, as well as periods spent in the Intensive Therapy Unit (ITU).
During his recovery period, George developed complications, including influenza and a chest infection, which led to him starting new intravenous antibiotics.
“Duchenne Muscular Dystrophy is complex, cruel, devastating, and unpredictable. George has undergone two enormous and high-risk surgeries within three weeks. It hasn't been straightforward, with a few hairy episodes,” the Naughton family said in an update on the Join Our Boys social media page.
“It has taken the skill of a phenomenal surgeon, Mr. Pat Kiely. We know how lucky George is to get this opportunity, and we are very, very lucky to have the best surgeon and team.”
He is currently receiving hospital treatment.
The family extended sincere thanks to everyone at Children’s Health Ireland for their continued care and support, along with the numerous professionals who have lent their skills.
“We can never truly explain the skill, care, support, and love George continues to receive. The service is incredible. The staff work in a very busy and stressful environment, yet somehow manage to navigate it all with such skill, dignity, grace, humour, and patience,” they said.
“They are truly remarkable clinicians, nurses, physios, OTs, dieticians, social workers, administrators, care staff, and support staff.
“We will never, ever be able to thank them enough for what they have done for George, Isaac, and thousands of other children in this state. They transform lives and save lives every single day. We remain indebted to them all.”
The Naughton family also expressed their gratitude to everyone who has supported them, whether through kind words or donations.
'Sweet 16' Campaign
The Naughton family recently launched the Sweet 16 campaign.
One project within this campsign aims to bring 1.6k people together for a the VHI 10k mini marathon event.
Taking place on Sunday, May 31, in Dublin, the fundraising event marks a poignant milestone for the boys, as they approach their 16th birthday.
If you would like to commit to take part for the join the boys trust, please email, join@determend.com or ring/text: 0879225999. There is an option of attending in person or virtually.