Lectureship to develop clinical trial agenda
A consortium of charities working on Duchenne muscular dystrophy (DMD) has awarded a lectureship to Dr Michela Guglieri in partnership with Newcastle University and NUTH to develop and advance the clinical trial agenda for this devastating and currently incurable condition. The group of charities, Action Duchenne, Alex’s Wish, Duchenne Children’s Trust, Duchenne Now, Duchenne Research Fund, Harrison’s Fund, Joining Jack have a shared goal to bring new treatments to the clinic for Duchenne muscular dystrophy as quickly as possible.
The award of £250,000 over five years is being matched by Newcastle University to enable Dr Guglieri to develop the clinical trial capacity for DMD at the John Walton Muscular Dystrophy Research Centre and nationally via work on a range of different projects which will begin to address the clinic trail capacity gap that we currently face. This lectureship will therefore consolidate knowledge on the best way to run trials for DMD. There are currently eight trials in DMD ongoing at the Clinical Research Facility at NUTH supported by the John Walton Muscular Dystrophy Research Centre and by the NIHR CRN. Several new studies are also on the horizon.
Duchenne muscular dystrophy (DMD) is a severe form of muscular dystrophy, characterised by progressive muscle weakness and wasting affecting all muscles in both legs and arms. DMD is caused by a mutation (fault) in the dystrophin gene. This gene encode of the protein dystrophin, which is an important component of the muscles. Boys with DMD do not produce any dystrophin and this leads muscle fibres to break down and be replaced by fibrous and or fatty tissue. Affected children usually develop the first symptoms of weakness at the age of 2 to 4 years with frequent falls, inability to run and jump, difficulties climbing stairs and getting up from the floor. As the condition progresses boys with DMD are unable to walk as far or as fast as other children. The muscle weakness continues to progress and without treatment boys usually lose the ability to walk independently between the age of 8 and 12. The heart and the respiratory muscles are also affected leading to a reduce life expectancy. There is no cure for Duchenne muscular dystrophy available so far. Steroids can delay the progression of symptoms but do not change the natural course of the condition. Intensive research to find a cure is carrying on in many centres around the world to try to find a way to induce the muscles to produce dystrophin or get round the effects on the gene fault.
About the John Walton Muscular Dystrophy Research Centre
The John Walton Muscular Dystrophy Research Centre brings together and consolidates Newcastle’s distinguished, international and world-leading record in research and care for neuromuscular diseases.
The team of over 70 people based at Newcastle University and its associated hospitals work together towards the development and application of genomic and translational medicine to improve the health outcomes of people living with neuromuscular diseases.
As a group, the Centre has developed a close and important link between research and clinical activities and actively pursues new partnerships with other stakeholders such as patient organisations, regulators and pharma.
The John Walton Centre’s key areas of activity – including clinical care, research, diagnostics and networking – all are strongly interlinked. It is the collaboration and work across these teams that are key to the Centre’s success and impact in the neuromuscular field.