Policy and campaigning

House of Lords Science and Technology Committee inquiry: The impact of Brexit on UK Duchenne muscular dystrophy research

Duchenne UK has submitted evidence to the House of Lords Science and Technology Committee inquiry into Life Sciences and the Industrial Strategy.

Duchenne UK believes that Brexit poses challenges to progress in rare disease research, access to treatment and scientific collaboration.

The Life Sciences Industrial Strategy outlines how the government can make the UK the best place in the world to invest in life sciences. The strategy will inform the Sector Deal – the agreement of what both industry and the government will do to achieve this. The strategy comes 14 months after the UK’s decision to leave the European Union.

The House of Lords will scrutinise the government’s approach to the Life Sciences Strategy. They will investigate whether the Government has the necessary structures in place to support the life sciences sector; how the NHS can make the most of new medicines and innovations, and the challenges and opportunities posed by Brexit.

Duchenne UK has outlined to the Lords the benefits of EU collaboration to the life sciences, and particularly muscular dystrophy and other rare diseases. It is imperative that these benefits are not lost or overlooked in the Brexit negotiations.

Key points from Duchenne UK’s submission include:

  • Patients in the UK have access to potentially life-saving clinical trials due to EU funding. In five years, more than €22million has been invested in Duchenne muscular dystrophy research through the EU’s FP7 and Horizon 2020 programmes. In total, they invest €150 billion in research and innovation. Leaving the EU will put access to future funding in jeopardy.
  • The UK is facing a skills shortage – recruiting doctors to train as clinical research associates (CRAs) and specialise in running neuromuscular trials remains a significant challenge. Many CRAs are recruited from outside the UK and Brexit places uncertainty over their future in the UK.
  • Brexit risks placing the UK in a silo by inhibiting its ability to work with the EU. The UK is internationally renowned in DMD research, but Brexit has the potential to stifle the UK’s contribution to the international scientific and medical communities.
  • Loss of involvement in the EU’s regulatory pharmaceutical framework could make the UK a less attractive place for pharmaceutical companies to launch new drugs, and conduct clinical trials. This will directly impact on patients’ access to the latest life-saving drugs.

You can read Duchenne UK’s Written evidence to the House of Lords Life Sciences and the Industrial Strategy inquiry here.

You can read Duchenne UK’s response to the Life Sciences Industrial Strategy here. 



About Duchenne Muscular Dystrophy (DMD)


  • There are around 2,500 boys affected by DMD in the UK and around 300,000 worldwide. It is classified as a rare disease
  • Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood, almost exclusively affecting boys
  • Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. There is no treatment or cure


About Duchenne UK


  • Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy (DMD). Duchenne UK has raised more than £5.5million in five years, and spent or committed £5million to DMD research projects – more than any other organization
  • Duchenne UK is leading the drive towards the efficient repurposing of existing medicines to bring new treatments for DMD
  • Duchenne UK believes that every child with Duchenne should be given the opportunity to access a clinical trial. Duchenne UK created the DMD Hub in 2016 to expand and develop the infrastructure for DMD trials in the UK and accelerate access to clinical studies for those who are interested in participating
  • The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell



Visit duchenneuk.org for more information or follow @DuchenneUK on Twitter.


Contact or more information and interview requests: Molly Hunt, Communications Manager: [email protected]


Published on 10 November 2017

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