Duchenne UK and Leeds Hospitals Charity have partnered to fund two new roles within the Neuromuscular Service over the next five years. Together, they are contributing more than £300,000 to support this specialist team, recognising its status as a centre of excellence in paediatric neuromuscular research.
As part of the service, NIHR Leeds Clinical Research Facility (CRF) Children’s Site delivers pioneering clinical trials, exploring innovative treatments for neuromuscular conditions at Leeds Children’s Hospital and has consistently led the way in this area of research.
The NIHR Leeds CRF has been the first site in the UK to recruit to several clinical studies for Duchenne muscular dystrophy (DMD), the most common and severe form of childhood muscular dystrophy. Most recently, the children’s research unit delivered a landmark Phase 1 trial investigating a promising new treatment for DMD.
11-year-old Austin was diagnosed with the condition in November 2015, at just two years old. Austin’s mother Maxine initially thought he had a virus and took him to hospital, where he spent the next month having tests.
Three years ago, Austin began a trial at the NIHR Clinical Research Facility Children’s Site. Austin has received over 130 infusions, and though he knows he will lose the ability to walk, he tells Maxine he takes part in the trials because, ‘I want to help boys in the future to walk longer.’
For Austin’s family, the opportunity to participate in clinical trials has been a lifeline, offering hope to other families to find better treatments and eventually a cure. Austin’s current trial ends in September, but Maxine says if he chooses to continue to take part in research trials, they will continue to visit hospitals up and down the country.
‘From the moment Austin was diagnosed, our world changed. Nothing could have prepared me for the grief of knowing his life would be cut short. But through the darkness, clinical trials gave us hope. The support from the team in Leeds has been incredible; they’ve become like family. Research is vital, not just for medical breakthroughs, but for families like ours to feel less alone. Austin is paving the way, and we’re so proud of him.’
The funding from Duchenne UK and Leeds Hospital Charity will go towards funding the role of Consultant Paediatric Neurologist Dr Anne-Marie Childs. Dr Childs said the funding would be enable the service to plan for the future.
‘We’re incredibly grateful to both Leeds Hospitals Charity and Duchenne UK for their grant funding to support the children’s neuromuscular service. This investment will allow us to attract the best people to work in the Leeds neuromuscular team and create new opportunities for clinical trials that can support even more families. Neuromuscular conditions have a devastating impact, not just on the child affected but the whole family; funding like this gives hope that new treatments and new drugs can be developed.’
