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Dublin Live
2 days ago
- Health
- Dublin Live
Devastated Dublin mum fights for life-changing medicine to be approved for son
Our community members are treated to special offers, promotions and adverts from us and our partners. You can check out at any time. More info 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. Archie Ennis, 8, from Tallaght, suffers from Duchenne muscular dystrophy and is in desperate need of medication to help his condition. His mother, Una Ennis, said that "time is muscle" and children like Archie could face dying in their 20s if medicine is not approved quickly. Archie 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. (Image: Una Ennis) 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. (Image: Una Ennis) "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 Fail 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." Join our Dublin Live breaking news service on WhatsApp. Click this link to receive your daily dose of Dublin Live content. We also treat our community members to special offers, promotions, and adverts from us and our partners. If you don't like our community, you can check out any time you like. If you're curious, you can read our Privacy Notice. For all the latest news from Dublin and surrounding areas visit our homepage.
Irish Daily Mirror
2 days ago
- Health
- Irish Daily Mirror
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."
The Independent
28-02-2025
- Health
- The Independent
Children denied access to life-changing drug due to paperwork, say families
The families of children with a severe genetic muscle-wasting condition claim NHS failings are preventing them from accessing a drug that slows its progression. Clinical teams lack the 'capacity to process the paperwork' associated with administering givinostat, campaigners said. The drug was made available to eligible patients through a type of compassionate use scheme known as an Early Access Programme (EAP) last year. Parents described the situation as 'unfathomable' and called for immediate action. Duchenne muscular dystrophy (DMD) is the most common and severe form of muscular dystrophy. It causes muscle degeneration and weakness due to a mutation in the gene that produces dystrophin, a protein that protects muscle fibres from breaking down. The condition usually only affects boys, with patients unable to walk by the age of 12 and usually only surviving into their 20s and 30s. There are about 2,500 people in the UK living with DMD at any one time, with about 100 new diagnoses each year. Givinostat, also known as the brand name Duvyzat, is a first-of-its-kind treatment that belongs to a family of drugs known as histone deacetylase (HDAC) inhibitors. These treatments reduce inflammation and fibrosis in muscle tissues while promoting muscle regeneration and slowing down the progression of DMD. An EAP opened for givinostat at the end of 2024, meaning its manufacturer has made it available free of charge to the health service until regulators make a decision about its cost effectiveness and availability for routine access through the NHS. Patients over the age of six with a confirmed diagnosis of DMD are eligible. They should also be able to stand for less than 10 seconds, and should have been on a stable corticosteroid for at least six months prior to start the treatment. Participation in the EAP is decided at an individual NHS trust level. However, the charity Duchenne UK, which was founded by two mothers whose sons have DMD, claims no NHS trusts currently offer givinostat. It alleges families have been told trusts do not have the resources to process the paperwork needed to administer the drug, or conduct blood tests for monitoring. The charity's co-founder Alex Johnson, whose 17-year-old son Jack was diagnosed with DMD in 2011, said: 'When it was announced in November that givinostat was going to be available at no cost to patients or the NHS, families like ours were understandably excited. 'This is the first time there has been a treatment that can slow down the progression of the condition, so it is a huge moment for us. 'To later find out that the life-changing treatment – which is being given to the NHS for free – cannot reach the people who so desperately need it, all because clinical teams don't have the capacity to process the paperwork, is unfathomable.' Co-founder Emily Reuben, whose son Eli, 17, also has DMD, added: 'Pressures on the healthcare system are currently failing our children. 'We can't afford to sit back and accept that, because every second counts when it comes to Duchenne muscular dystrophy. 'That is why we're calling for support to make sure that more time is not lost.' In January, the charity's co-founders and other families met with MPs, including Health Secretary Wes Streeting, to highlight the issue. Ms Reuben said: 'We're really pleased that Wes Streeting and his colleagues spent so much time chatting to us and our boys and hope to work together to ensure that NHS trusts can overcome these challenges to help us to give our boys a fighting chance.' A Department of Health and Social Care spokesperson said: 'We are committed to supporting access to specialist care, treatment and drugs for those with rare diseases. 'It is for individual trusts to decide whether to take part in an Early Access Programme which provides access to givinostat.' NHS spending watchdog the National Institute for Health and Care Excellence is currently appraising givinostat to determine if it should be rolled out on the health service. A decision is expected later this year. The drug was approved by the Medicines and Healthcare products Regulatory Agency in December. An NHS spokesperson said: 'We know how important access to new treatments is for patients and their families living with progressive conditions like Duchenne muscular dystrophy. 'So while Nice determine whether givinostat should be made routinely available and funded on the NHS, a number of trusts across the country are in the process of making preparations to offer the treatment via a company-led Early Access Programme, in which trusts themselves must cover substantial costs to administer the treatment.'
