5 days Ago By [email protected] (Sam Volpe)
The mum of Gateshead brothers who have a rare genetic condition has paid tribute to the "life-changing" care they get at Newcastle's pioneering John Walton Muscular Dystrophy Research Centre. The pair - Freddie and Louis Tanner-Boyer - are aged six and three respectively and they both have spinal muscular atrophy (SMA) which causes muscle weakness, and can lead to mobility and even breathing issues. Freddie uses a wheelchair and has been given a groundbreaking drug called Nusineresen, while receive care at the John Walton Muscular Dystrophy Research Centre.
Mum Steph, 33, is a specialist nurse and she's been so inspired by the care her boys have received that she now works at the John Walton centre , which is run as a collaboration by Newcastle University and the Newcastle Hospitals NHS Trust. She said: "She said: “We feel so lucky that Freddie and Louis receive care at the centre because the treatment they have both received has been life-changing and we’re very thankful for that. "The centre is a leader in its field - the expertise of the specialists working there is outstanding and the breakthroughs they continue to make offers hope to patients.
" The John Walton centre - based at the Centre for Life - is marking its tenth birthday and also celebrating news that another drug developed in Newcastle has now been approved. That is called Vamorolone, which is specifically for Duchenne muscular dystrophy (DMD), a paediatric muscle wasting disease mainly affecting boys. Professor Michela Guglieri was awarded an EU Horizon 2020 grant worth £6 million to develop this as a safe and effective therapy for all patients living with the condition.
It is a corticosteroid and seen as a "significant improvement" on previous treatments. Professor Volker Straub, centre director, said: “I am very proud that we are celebrating the 10-year anniversary of the research centre. Our team is from a variety of backgrounds and works on a comprehensive programme of patient-centred clinical and research activities to further advance understanding of rare diseases, focussing on neuromuscular problems.
"We have been a key part of several medical breakthroughs over the last decade, such as the development of transformative drugs for spinal muscular atrophy, and the starting of UK-first trials of gene therapy for Duchenne muscular dystrophy. I’m excited to see what further scientific advances we can continue to make over the next 10-years." Sir Jim Mackey, chief executive at the Newcastle Hospitals NHS Trust, said: "The John Walton Muscular Dystrophy Research Centre has been at the forefront of neuromuscular research and scientific breakthroughs over the last decade.
"It brings us great pride to see the centre is leading the way in clinical trials to find much-needed treatments for neuromuscular conditions, which we know will have a significant impact on patients and their families." Join our Breaking News and Top Stories WhatsApp community for all the latest news direct to your phone. To join you need to have WhatsApp on your device.
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