Latest news with #AddyClarke
Yahoo
7 days ago
- General
- Yahoo
Swindon family of girl with rare disease 'horrified' at latest trial update
The Swindon family of a young girl with a rare brain disease welcomed the news that her trial of lifesaving treatment will continue indefinitely. Addy Clarke is just one of 100 children in the world to be diagnosed with Batten Disease (CLN2), which is gradually taking away her ability to talk, eat and walk independently, and see. Her parents Hayley and Dave have previously spoken to the Adver about their heartbreak at seeing their daughter fade before their eyes while she is supported by vital cerliponase alfa treatments. Earlier this month, they celebrated NICE and the NHS coming to a commercial agreement to continue that treatment for Addy and all other children with CLN2 after the trial ends in 2025, describing the news as 'amazing'. But they were saddened to learn that the same access to the treatment would not be given to any child diagnosed after January 1, 2026. Recommended reading Building 700 homes at old golf course 'best option' for the area Family pays tribute to cyclist killed in collision by Wiltshire driver Wedding venue can continue to host events after planning appeal NICE explained that although its committee took into account the condition's rarity, severity and the effect of the treatment on quality and length of life, the most likely cost-effective estimate based on the proposed price of the medicine is not within what it considers to be an acceptable use of NHS resources. In a statement on Addy's Batten Adventure Facebook page, the Clarke family said: 'Whilst we are happy for our own little Addy, we sadly know firsthand the devastation that this news can cause on a family - which would be made all the worse if you knew other children in the country were on the same treatment that your child couldn't access because of the timing they were diagnosed, and cost. 'For us, this treatment has never been simply just about our Addy, but all children diagnosed with this earth-shattering disease - therefore we will fight on. 'The decisions by NICE have been horrific and it feels like they are trying to manipulate the pharmaceutical companies by using families as pawns in game. 'Thank you for all your support you have shown us through the ongoing questions, the donations, prayers, meals and many other things over the past five years of this journey. 'We take a big deep breath, gather our thoughts and go again.' The family has met with South Swindon MP Heidi Alexander as they try to raise awareness of this disparity in the treatment of children with the rare disease. As for Addy herself, she recently celebrated her ninth birthday by having a party with her friends and classmates at Swindon's hydrotherapy pool.
BBC News
16-05-2025
- Health
- BBC News
Swindon girl with Batten Disease will continue to get vital drug
A family has been told their child will continue to get access to a drug which she began taking as part of a trial, and is helping to keep her Addy Clarke, from Swindon, has Batten Disease - a rare degenerative disease that has no agreement has been reached that existing patients will continue to have the drug Brineura, which costs £500,000 per patient per year, via the it has not been recommended for future patients "due to its high price and the limited evidence of long-term effectiveness", said the National Institute for Health and Care Excellence (NICE). Addy's mother Hayley Clarke said she is "absolutely thrilled and relieved and so thankful" at the news but wants to get access to the drug for other children. "There's still another half a fight to go to get it secured for all children, future children most importantly," she said the family could have lost Addy by now, that she should be blind, but her eyesight has been preserved and she can still make herself of the main symptoms of Batten Disease is childhood dementia. "We're just so thankful for the time that this drug is giving us with her," said Hayley, who also told the BBC about her daughter's passions for lions and swimming."I think I'm still processing that it's a yes for our daughter," she added."[I'm] almost trying to be really guarded with hope because we've had too much shattered hope along the way."Hayley said the family has had a lot of support from people around them as well as local hospice charities and the council. The family went with other with members with Batten Disease to Westminster to ask for continued access to guidance from NICE did not recommend using it for future patients due to cost and long-term effectiveness, but said it had reached a deal with the NHS and manufacturer BioMarin for those already using it and anyone signed up by the end of 2025. Helen Knight, director of medicines evaluation at NICE said: "We know this is not entirely the news people in the Batten Disease community were hoping for. However, this is not the end of the story. We will continue to work with all parties towards a solution."The charity Batten Disease Family Association said it was pleased with the agreement, but said as the NICE guidance is a draft and not final, it will continue to push to make Brineura accessible to all children who need it.
