Accelerating access to treatments
We know that funding research alone is not enough to ensure that DMD patients can access the treatments they need. That's why we're going further, and breaking down barriers at every stage of drug development.
We know that funding research alone is not enough to ensure that DMD patients can access the treatments they need. That's why we're going further, and breaking down barriers at every stage of drug development.
DMD is a progressive disease, and we know that this generation of boys can't wait. That's why we've launched innovative, collaborative projects to increase the chances of treatments being tested in clinical trials, approved and made available to those that could benefit from them as soon as possible.
NICE has released draft guidance which does not recommend the use of ataluren on the NHS for the treatment of Duchenne muscular dystrophy (DMD) with a nonsense mutation in the dystrophin gene.
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Fleur Chandler is a health economist and currently Head of Market Access at Sanofi, a worldwide pharmaceutical and healthcare company. She is also mum to Dominic, a 16-year-old boy living with DMD.
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By funding staff posts at the Royal Hospital for Children, Glasgow, the DMD Hub has enabled more trials to take place and more boys in Scotland to take part in research
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NICE and NHS England have released a statement about continued access for DMD patients currently being treated with ataluren
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CEO Emily Reuben spoke at the ABPI conference and shared this thought piece on access to DMD treatments
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A new drug aiming to slow progression of DMD, Givinostat, has shown positive results in a clinical trial.
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Duchenne UK and the DMD Hub are launching a new pilot project aiming to provide fair and equal access to DMD trials across the UK
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In a new transatlantic collaboration, Duchenne UK and the DMD Hub, PPMD and RTI International are conducting a study to explore attitudes towards gene therapy and gene editing from adults with Duchenne muscular dystrophy (DMD), parents and caregivers of children with DMD, and clinicians.
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The Clinical Trials Lectureship grant enabled Dr Michela Guglieri to act as the Clinical Research Team Leader within the John Walton Muscular Dystrophy Research Centre in Newcastle
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We set up HERCULES, a collaborative project to increase the chances of DMD patients accessing treatments.
Visit the HERCULES site
We founded the DMD Hub to increase the number of UK sites able to run clinical trials for DMD.
Visit the DMD Hub site
Find out about the research we fund to find the most effective treatments for DMD.
Our research
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