Couple push for drug that slows down muscular dystrophy to be made available in Ireland
Dubliners Una Ennis and her husband Kenneth ran a high-profile campaign for their son Archie who was recently diagnosed with a muscle wasting disease that affects around 120 boys in Ireland.
The family has raised more than €1m to help secure specialist gene therapy treatment for Archie, which will cost €3.5million in the US.
In the meantime, Una Ennis is calling on the government to pay for a drug that is being rolled out in parts of Britain which will help slow down the disease that is attacking her son's body.
'I met with a number of TDs in Leinster House after we held a peaceful demonstration outside and we wanted to show a presence for these children," said Mrs Ennis.
'We are trying for Archie and for all the children to get this drug brought into Ireland to help them.
To get the diagnosis but have no medicine is heartbreaking, we need to have hope.
'The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it.
'We are still waiting on the pharmaceutical companies to put their application into the government to cover the costs or sign off on it.
"Either way there is no answer yet, but we have had a lot of support from lots of TDs including Sean Crowe and Senator Teresa Costello and progress is being made."
Givinostat is a drug used to treat muscular dystrophy in children while they can still walk. It works by slowing down the muscle deterioration by turning genes on and off within cells particularly in the muscle tissue.
The disease itself is a genetic disorder that weakens muscles and there is no cure. Una and Kenneth Ennis are now hoping to meet with the minister for health Jennifer Carroll MacNeill to discuss their son's situation.
'She has responded to us and is willing to meet with us, but we need the drug to be paid for as soon as possible. Time is muscle, that is the most important thing," said Una.
'Every day counts, we need this early access, there is an alternative in America but the treatment there is €3.5million.
'If we could just get that drug over the line, that would give all our children a start. But we need medicine now.
I can't watch my son get weaker. I think anyone with a child affected by this will know how we feel. They all deserve it.
'Time is muscle and all we can offer children here is a wheelchair, and physiotherapy which takes six months as well as some steroids.
'In the UK it is the next level, the standard of care there is unreal. We should have the same treatment here and not have to travel to the UK for it.'
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Irish Examiner
30-06-2025
- Irish Examiner
Couple push for drug that slows down muscular dystrophy to be made available in Ireland
The mother of an eight-year-old boy who suffers from muscular dystrophy has met with TDs to push for a new potentially lifesaving drug to be made available in Ireland. Dubliners Una Ennis and her husband Kenneth ran a high-profile campaign for their son Archie who was recently diagnosed with a muscle wasting disease that affects around 120 boys in Ireland. The family has raised more than €1m to help secure specialist gene therapy treatment for Archie, which will cost €3.5million in the US. In the meantime, Una Ennis is calling on the government to pay for a drug that is being rolled out in parts of Britain which will help slow down the disease that is attacking her son's body. 'I met with a number of TDs in Leinster House after we held a peaceful demonstration outside and we wanted to show a presence for these children," said Mrs Ennis. 'We are trying for Archie and for all the children to get this drug brought into Ireland to help them. To get the diagnosis but have no medicine is heartbreaking, we need to have hope. 'The drug Givinostat has been brought into parts of Britain. We want that too, but we need the government to pay for it. 'We are still waiting on the pharmaceutical companies to put their application into the government to cover the costs or sign off on it. "Either way there is no answer yet, but we have had a lot of support from lots of TDs including Sean Crowe and Senator Teresa Costello and progress is being made." Givinostat is a drug used to treat muscular dystrophy in children while they can still walk. It works by slowing down the muscle deterioration by turning genes on and off within cells particularly in the muscle tissue. The disease itself is a genetic disorder that weakens muscles and there is no cure. Una and Kenneth Ennis are now hoping to meet with the minister for health Jennifer Carroll MacNeill to discuss their son's situation. 'She has responded to us and is willing to meet with us, but we need the drug to be paid for as soon as possible. Time is muscle, that is the most important thing," said Una. 'Every day counts, we need this early access, there is an alternative in America but the treatment there is €3.5million. 'If we could just get that drug over the line, that would give all our children a start. But we need medicine now. I can't watch my son get weaker. I think anyone with a child affected by this will know how we feel. They all deserve it. 'Time is muscle and all we can offer children here is a wheelchair, and physiotherapy which takes six months as well as some steroids. 'In the UK it is the next level, the standard of care there is unreal. We should have the same treatment here and not have to travel to the UK for it.'
The Journal
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RTÉ News
11-06-2025
- RTÉ News
Families call for approval of new 'game changer' Muscular Dystrophy drug
Parents of children with an incurable muscle-wasting condition have called on the Minister for Health to accelerate the approval and funding of a new drug which they have described as a "game changer". It is estimated that over 100 boys are currently living with Duchenne Muscular Dystrophy (DMD), a genetic disorder, mainly affecting males, that causes progressive muscle weakness. As the life-limiting condition progresses, it affects the respiratory system and over time those with the condition may need the use of a wheelchair. Families affected are now campaigning for the approval of a new drug, known as Givinostat, to be accelerated. Among those calling on the medicine to be made available to patients here are the Brogans from Deansgrange in Dublin. Their nine-year-old son, Fionn, was diagnosed with DMD when he was aged three. 'We were told on the day of the diagnosis that there really wasn't many options of to go off and ''live your best life'',' said mother Meave Brogan. Ms Brogan said Givinostat is a "massive game changer". While it does not provide a cure for the condition, clinical trials, she says, have shown it can significantly slow down its progression. "There's no cure for Duchenne, but hope for a real slowdown of the progression." 'We've had to have a lot of really hard to him why he's struggling is the first time that we could have a conversation about something that could help," said Ms Brogan. "What makes it so difficult is that we have to explain to him that it's out there, but we just can't access it. "So the hope feels actually quite painful. It's so close, but it's still really far away because of the process that it has to go through." The drug received EU conditional marketing authorisation earlier this week, meaning it is on course for fast track approval as it fulfils an unmet medical need. Campaigners here are concerned that even with full approval, it could take up to two years before it is rolled out. "If it was anything near that, it's likely that Fionn will not be able to access that drug, and that is absolutely heartbreaking," said his dad, Michael Brogan. "But it's also unacceptable to us, and that's why we're calling on the Minister for Health to intervene." Mr Brogan said those with the condition do not have time to wait and that every day muscle is lost. 'We've a really resilient, really determined boy who wants to get on with life, and he wants to do the best he can we want to give him every opportunity to be able to do everything he wants to do in life," he said. "There's no cure for Duchenne, but hope for a real slowdown of the progression. "There's examples of boys that are still able to take steps in their late teens," he said. Families affected are gathering outside Leinster House to bring their campaign directly to politicians. They are also calling for the health authorities here to follow Scotland and England in speeding up approval. In a statement, the Department of Health said it was aware of the plight of patients with DMD and acknowledges the important of access to medicines. It said the HSE has statutory responsibility for decisions on medicines, based on comparative clinical and cost effectiveness and engages with patients and clinicians throughout the assessment process. It also said if the makers of the drug choose to submit a pricing and reimbursement application, the HSE will give "fair consideration" to the application.
