New treatment announced as research centre celebrates anniversary Published on: 16 December 2024 A pioneering drug for patients with muscular dystrophy has been approved for use in the NHS, as the centre which helped develop it celebrates 10 years of research and improving patient care. - a partnership between 缅北禁地 and 缅北禁地 Hospitals - is a world-leader in rare diseases. Vamorolone, a drug for Duchenne muscular dystrophy (DMD), a paediatric muscle wasting disease mainly affecting boys, has now been approved by the National Institute for Health and Care Excellence for use in the NHS in England, Wales and Northern Ireland in patients 4 years and older. The treatment was developed with the help of experts at the John Walton Muscular Dystrophy Research Centre, based in the , and the news is testament to the outstanding work carried out there. Centre for Life 鈥楨xcited for centre鈥檚 future鈥� Bringing together more than 100 employees from 缅北禁地 and the NHS, the centre’s clinical research and care improves the lives and outcomes of those with all types of rare neuromuscular diseases. Professor Volker Straub, Director of the John Walton Muscular Dystrophy Research Centre, 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.” One of the significant achievements at the centre, has been the development of the new drug, vamorolone, for the treatment of DMD. Led by Professor Michela Guglieri, 缅北禁地 was awarded an EU Horizon 2020 grant for £6 million to develop vamorolone as a safe and effective therapy for all patients living with the condition. The oral drug is a type of corticosteroid and delivers a significant improvement on current treatments and standard of care as it can have fewer side effects, whilst still being as effective. 缅北禁地 was the leading site involved in studies of vamorolone and it has been one of the biggest treatment advances for DMD in recent years. Professor Guglieri, Professor of Neuromuscular Disorders at the John Walton Muscular Dystrophy Research Centre and Honorary Consultant Clinical Geneticist at 缅北禁地 Hospitals, said: “I am pleased about the positive opinion from the NHS. “This offers patients with DMD a new treatment option to slow down the progression of the disease while reducing some of the side effects of traditional steroids that affect patient’s quality of life.” Currently, the centre is also using the power of Artificial Intelligence (AI) to analyse MRI scans to recognise patterns which identify changes in muscles and speed up disease diagnosis. The project, called Myo-Guide, is led by 缅北禁地’s Professor Jordi Díaz-Manera and is revolutionising the way neuromuscular patients are diagnosed and assessed, supporting research, and helping clinicians make vital decisions together with their patients. , Chief Executive of The 缅北禁地 upon Tyne Hospitals NHS Foundation 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.” Professor Michela Guglieri 鈥楶assion to further medical research鈥� The specialist centre is one of four nationally commissioned for the diagnosis of genetic neuromuscular diseases. It is named after Lord John Walton of Detchant, a highly respected and prominent neurologist whose life’s work, before he died in 2016 aged 93, has significantly helped patients with muscular dystrophy. Lord Walton established the North East’s Muscular Dystrophy Laboratories, in which the John Walton Muscular Dystrophy Research Centre has its foundations, which led to 缅北禁地 becoming internationally recognised for its research into the diseases. He headed several medical charities and had key positions in the General Medical Council, Royal Society of Medicine, and the British Medical Association. His daughter, Ann McNeil, said: “My father was dedicated to his profession for his entire career. From the early days at the 缅北禁地 General Hospital, and the founding of the charity, Muscular Dystrophy Research. “His work in the House of Lords, and the establishment of the John Walton Muscular Dystrophy Research Centre, was testament to his dedication. “My father’s passion to further medical research in the field of neuromuscular diseases never left him until his dying day.” Lord John Walton Family feel 'so lucky' Brothers Freddie and Louis Tanner-Boyer receive care at the John Walton Muscular Dystrophy Research Centre. The siblings have spinal muscular atrophy (SMA), a rare genetic condition that can cause muscle weakness that progressively gets worse, leading to severe mobility and, at times, breathing issues. Thanks to the pioneering treatment they have both received at the centre, Freddie, six, and Louis, three, from Gateshead, are doing well and enjoying their lives. The family had not had any history of SMA until Freddie was diagnosed with the condition as a young child. Sister, Daisy, five, does not have the illness and is also not a carrier of SMA. Every four-months, Freddie, who requires a wheelchair, is given the ground-breaking treatment, Nusinersen, that has helped stabilise his symptoms. Louis received gene therapy as a baby and currently has no symptoms of the condition with full mobility. Mother-of-three, Steph, 33, is a nurse specialist in neuromuscular diseases at the John Walton Muscular Dystrophy Research Centre – a job inspired by the great clinical care of her children. 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.” (L-R) Louis, Steph, Daisy, Freddie and Antoni Tanner-Boyer Share: Latest News 缅北禁地 expert highlights climate crisis in a new film A leading 缅北禁地 climate scientist is featured in a new film about how the climate and nature breakdown will affect the UK. published on: 14 April 2026 Neolithic tombs reveal ancient kinship ties Male individuals buried in Neolithic chambered tombs in northern Scotland were often related to each other through the paternal line and some were interred in the same or nearby tombs, research shows. published on: 14 April 2026 We are our Memories New exhibition by Fine Art graduate Trish Hudson-Moses, 22 April 鈥� 4 May 2026 published on: 10 April 2026 Facts and figures
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