Latest news with #EarlyAccessProgramme
Daily Mirror
5 days ago
- Health
- Daily Mirror
'My son, 12, can't have free life-changing drug because of where we live'
No mother should have to watch their child suffer, but one woman shares what it's like witnessing her 12-year-old boy slowly deteriorate because he's not eligible for a new drug that could improve his life Having to watch her son slowly fade away is heartbreaking in itself but knowing he can't have a free drug because of where he lives, is nothing less than mental torture. That's the everyday reality for Colleen who is mum to Alfie Pentony, 12, from Newry, County Down, who was diagnosed with Duchenne Muscular Dystrophy (DMD) when he was just four years old. Her fun-loving, football -mad son is getting weaker every day - but a new drug, Givinostat, could slow down his incurable condition and help him walk for another three years. But although the drug is free in other parts of the UK, the Belfast Trust has said it does 'not have the capacity' to offer it in Northern Ireland. 'We're heartbroken and devastated,' Colleen, 43, tells The Mirror. 'It's unjust, shameful and feels like mental torture knowing there's a drug that could help him but he can't have it because of where we live. "Alfie is a typical little boy who wants to grow up and play for Newcastle United like Lewis Miley. We know this is never going to happen but if he got this drug, it would at least help prolong his ability to walk for maybe another three years and continue to kick a ball for longer.' Muscular Dystrophy is an inherited genetic condition that gradually causes the muscles to weaken so sufferers eventually lose the ability to walk - with most only living to their 20s or 30s. While DMD is one of the most common and severe forms - this new drug could slow it down giving patients an extra three years without being restricted to a wheelchair. 'We don't know how long Alfie has left on his feet, most boys are wheelchair bound at the age of 12, so he is beating the odds so far,' Colleen adds. 'His muscles are breaking down every day - but time is muscle and in a few months it could be too late for him. "Every day that he doesn't have access to the drug, he is getting weaker than the boys who have access to the drug which is unjust and cruel. The Trust has put a price tag on my son's life and I'm not having it, I don't know how they can sleep at night. 'This drug is something that could dramatically change the course of our son's disease and to be told we can't have it is mental torture. I feel let down by the service that is meant to protect these boys, the fact that we are the only part of the UK that won't provide this drug is shameful. It could massively prolong Alfie's life and his quality of life.' Alfie is also pleading for the drug - not just for himself - but for the 10 other boys in Northern Ireland who it could also help. The anti-inflammatory drug, Givinostat, has been approved for use in the UK by the medicines regulator and is available on the NHS for people who meet strict criteria. But individual trusts must apply to take part in the Early Access Programme (EAP). The Belfast Trust said currently it 'is not in a position to proceed' to offer the free drug. 'Its implementation will need to be managed within defined and agreed protocols and additional staffing resources will also be required to ensure the treatment can be provided safely," a spokesperson added. "We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them.'
Hans India
24-04-2025
- Hans India
Meta Adds Live Translation and New Features to Ray-Ban Smart Glasses
Meta is levelling up its Ray-Ban smart glasses experience by rolling out severalnew features and style options to a global audience. Among the biggest updatesis the availability of live translation for all users, previously limited toselect regions through Meta's Early Access Programme. Firstteased during Meta Connect 2024, live translation is now fully integrated intothe smart glasses. It allows users to communicate in English, French, Italian,or Spanish and hear real-time translations in their preferred language. Best ofall, if you download the relevant language packs in advance, the feature workseven without an internet connection—perfect for travellers on the go. In termsof aesthetics, the Ray-Ban Meta Skyler frame is now offered in a sleek, shinychalky grey paired with sapphire Transitions lenses. Meanwhile, the shiny black frames can be customised with clear or green-tinted lenses, offering more personalisation for fashion-forward users. Meta isalso preparing to expand several other features. Its Live AI capability—whichallows the Meta AI assistant to respond based on what you're currentlyseeing—will soon be available more broadly in the U.S. and Canada. This featureaims to enable more natural, context-aware interactions. Additionally,users will soon be able to send and receive direct messages, voice notes,photos, and even make Instagram calls directly through their glasses. Thisbuilds on existing support for WhatsApp, Messenger, and the default messagingapps on iOS and Android devices. Meta isgradually broadening its ecosystem for music lovers as well. Access toplatforms like Spotify, Apple Music, Amazon Music, and Shazam is starting toextend beyond North America. However, asking Meta AI to play a song or identifymusic remains exclusive to users with English set as their default language. In a moveto reach more global users, Meta has confirmed that its smart assistant, MetaAI, is now available in the UK and will roll out across all supported EUcountries by next week. Looking ahead, the Ray-Ban smart glasses are also setto launch in new markets including India, Mexico, and the United Arab Emirates,though no specific launch dates have been confirmed yet. Withthese enhancements, Meta is clearly aiming to make its smart glasses a morepowerful, stylish, and globally accessible wearable tech companion.
Yahoo
11-03-2025
- Health
- Yahoo
'He talked about dying... how do you even answer that?'
The mum of an 11-year-old boy who is waiting to find out if he can access a new drug to treat his Duchenne muscular dystrophy (DMD) said her son talked about his fear of death. Harry, from Corby, Northamptonshire, was diagnosed with the incurable condition when he was six. His mother, Rosemary, said "he's talked about being scared of dying, which is heartbreaking, how do you ever answer this as a parent?" University Hospitals Birmingham NHS Foundation Trust, which is treating Harry, has been contacted for comment. There are many types of muscular dystrophy which gradually cause the muscles to weaken, leading to an increasing level of disability. DMD is one of the most common and severe forms, usually affecting boys in early childhood. Many people with the condition will only live into their 20s or 30s. Harry's family believe the drug Givinostat could slow his decline, but said the hospital trust had not yet reached a decision on whether to offer the drug. Harry's Dad, Stephen, said having access to the new drug would give him "an extra two years of mobility and an extra two years of life would come along with that". Mum Rosemary added: "This is it. Once he loses those muscles and he can't walk and he is in a wheelchair, there is no coming back from that, he will never walk again. "Time very much is muscle, because every day he is getting weaker." Harry's older sister Lucy, 13, also hoped Harry could access the drug. "I can't imagine Harry being in a wheelchair or not being able to walk," she told the BBC. "Just the thought of that makes me upset. "I think it would be just amazing to see Harry really happy." Emily Reuben, chief executive and co-founder of the charity Duchenne UK, said: "Families across the country are asking for a free drug, which patients in Europe and the US are already accessing, that could keep our children living longer. "Time is muscle, and we are urgently campaigning to convince hospitals to provide access to this free medicine." The Medicines and Healthcare products Regulatory Agency (MHRA) said it granted conditional marketing approval for the drug in December. Interim executive director for healthcare quality and access Julian Beach said: "While the MHRA is not responsible for the relationship between the Early Access Programme and the NHS, we will support the healthcare system as needed to facilitate the process." Follow Northamptonshire news on BBC Sounds, Facebook, Instagram and X. 'It's cruel that I cannot get muscular dystrophy drug' Drug to benefit son delayed by bureaucracy, dad says Duchenne UK
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.'
BBC News
18-02-2025
- Health
- BBC News
Muscular dystrophy: Drug not being made available for children is 'cruel'
A 12-year-old boy with a severe form of muscular dystrophy has said it is "cruel" that a new drug is not being made available in Northern from Newry, County Down, was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years dad Jamie Pentony said the new drug, Givinostat, could slow his son's incurable condition down, but the Belfast Trust did "not have the capacity" to offer Belfast Trust said it was not in a position to proceed with the implementation of Givinostat as "additional staffing resources would be required to ensure the treatment could be provided safely". There are many types of muscular dystrophy. They are inherited genetic conditions that gradually cause the muscles to weaken, leading to an increasing level of is one of the most common and severe forms - it usually affects boys in early childhood and many people with the condition will only live into their 20s or on BBC Radio Ulster's Nolan Show, Mr Pentony said there were 13 boys in Northern Ireland who could benefit from the drug."Who is going to tell the 13 kids that could avail of this that some of them might not be walking next year?"Time is muscle when it comes to Duchenne. Your lung is a muscle, your heart is a muscle and most of the boys die from heart failure."They [the Belfast Trust] are telling us they do not have the capacity, they do not have the nurses to take the tests, that they do not have the neurologists to look after the boys whilst on this drug." Alfie said his condition could stop him from walking, but doctors have told him that because he was "doing so well, it will take a while for that to happen"."The drug would really help me, it would help me a lot, but there are other children that will stop walking before me, so it's important for them to get it as well," he said."It's cruel they are not giving this drug to kids in Northern Ireland with this disability. We all have the right to have something that could help." The anti-inflammatory drug, Givinostat, has been approved for use in the UK by the medicines regulator and is available early for people who meet strict criteria, on the trusts must apply to take part in the Early Access Programme (EAP).The Belfast Trust said: "The drug is available for an Early Access Programme (EAP) for eligible patients, pending decisions about wider access following the National Institute for Health and Care Excellence (NICE) recommendations."Currently, Belfast Trust is not in a position to proceed under the EAP. We recognise this will be very disappointing for families and the Belfast Trust sincerely apologises to them." 'Time is muscle' Una Farrell, from the charity Duchenne UK, said: "It's just hard to understand with a disease that is so cruel and progressive, and time is muscle, why children cannot be given access to it now."We understand that resources are a huge issue, but it's been known for several years that this drug was on its way."There is no cure for muscular dystrophy - finally there is a treatment that the data suggests can slow down the progression." Routine health service access to Givinostat will depend on guidance from health assessment body Ireland's Department of Health said it fully understood that patients and families want to access the best available health service treatments and drugs."The department maintains a formal link with NICE. In practice, this means that treatments that have been recommended by NICE for routine use in the NHS in England are also routinely available in Northern Ireland."NICE are developing guidance on the clinical and cost effectiveness of Givinostat for the treatment of DMD in people six years and over, with a committee meeting scheduled for May 2025."
