Mum urges Irish approval for medicine for son with life-limiting condition
A desperate mother has encouraged the State to quickly approve and fund life-changing medicine for her eight-year-old son who suffers from a life-changing and life-limiting rare form of muscular dystrophy.
Alfie Ennis suffers from Duchenne muscular dystrophy and is in desperate need of medication to help his condition.
His mother, Una Ennis, told the Irish Mirror that "time is muscle" and children like Archie could face dying in their 20s if medicine is not approved quickly.
Archie Ennis, 8, from Tallaght, south Dublin, was diagnosed with Duchenne muscular dystrophy in December 2024. The condition affects around 120 children in Ireland.
Mrs Ennis described Archie as a "little character" who loves to play with his friends and "be with all the boys".
He is aware his muscles get tired but he is a "determined" boy, who is now on steroids.
Mrs Ennis explained: "Archie was diagnosed with Duchenne muscular dystrophy two weeks before Christmas.
"We had a feeling something was going on with Archie. We actually thought at the start that it was just dyspraxia.
"I just thought he needed physio, but the physio wasn't helping, and she said, 'We'll go to the doctor'.
"[The doctor] said that Archie has Duchenne muscular dystrophy. It's a progressive muscle weakening condition which will cause his legs to become weaker and then his arms.
"She said that most boys who would have Duchenne would end up in a wheelchair.
"To hear that and to see our perfect little boy, our whole world was just turned upside down in an instant."
She went on to say that Archie's lungs will become weaker and his heart will become weaker.
"Most boys in their early 20s can die from cardio myopathy, which causes the muscles in the heart to become a lot weaker," she said.
"As parents, you're just devastated and desperate to try and get him some help.
"They say it's a rare condition, but it's the most aggressive muscular dystrophy.
"You hear all these stories of young boys passing away because they don't get medicine."
In April, the European Medicines Agency (EMA) recommended granting conditional marketing authorisation to the drug Duvyzat, also known as Givinostat, as a treatment for Duchenne muscular dystrophy.
Last week, the European Commission granted conditional market authorisation for the product.
However, Mrs Ennis now fears that it could take several years for Ireland and the National Centre for Pharmacoeconomics (NCPE) to approve its use on the Irish market.
She will hold a demonstration outside Leinster House on Wednesday afternoon before presenting to politicians in the AV Room from 2.30pm where she will tell them how urgent the medicine is.
Mrs Ennis continued: "Every day that Archie doesn't have medicine, his muscles are dying.
"If it's a year or two down the line [that the medicine is approved], I can't reverse that.
"I need the medicine as quick as possible, because time is muscle for Archie and all the boys with Duchenne."
In addition to Givinostat, Archie will require gene therapy. A doctor in Los Angeles has said that they can give him this treatment.
However, his parents have to fundraise €3.5m in order for him to travel to the US. Some €3.2m is required for the medicine, while the extra money is needed for the family to stay there for 12 weeks for the infusion. To date, over €800,000 has been raised.
In the meantime, Mrs Ennis is begging the Government and the NCPE to approve Givinostat to help Archie.
She is also urging Health Minister Jennifer Carroll MacNeill to attend the briefing in Leinster House on Wednesday.
Fianna Fáil Senator Teresa Costello has also urged the Minister to meet the affected parents.
The Tallaght-based senator said she emailed the Givinostat manufacturer last week as soon as progress was made with the European approval, asking them to expedite the time the medicine would be available to Irish children.
She added: "I will be watching the progress of this like a hawk because we just don't have two years to wait for this to be approved.
"Parents are in a really dire situation where every month there is a deterioration with their children."
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