Policy and campaigning

An update from PPMD's advocacy conference and gene therapy policy forum

On Sunday and Monday this week, our co-founders of Duchenne UK Alex Johnson and Emily Crossley went to Washington DC to attend Parent Project Muscular Dystrophy's annual advocacy conference and gene therapy policy forum. Here’s an update from Alex.

We would like to say a big thank you to Parent Project Muscular Dystrophy forallowingus to join their annual advocacy conference. We are grateful to you and all the patients and caregivers in the US who gave up their time to travel to Washington and meet their Congressmen and women and Senators, and to advocate for the Duchenne community. Their efforts created the MD Care Act which I believe has had a huge impact not just in the US but for the whole Duchenne community across the world. Listening to how your advocacy has helped to spur research breakthroughs, extend lifespan, improve the quality of care for those diagnosed with Duchenne and helped get the first therapies approved for Duchenne in the US was inspiring. Emily and I have talked extensively about how we can use these learnings to help our community here in the UK more effectively.

The Gene Therapy Policy Forum examined a range of issues and questions around the potential opportunities and challenges of gene therapies. Topics covered included the current status of trials, regulatory approval pathways, and the future of alternative delivery methods. These were explored with regulators, researchers, caregivers and patients. The discussions have helped us to think about how we can help support gene therapy trials in the UK through the DMD HUB, and the areas of research we could help fund in the future to overcome the challenges we face e.g. redosing, manufacturing supply and the potential responses of gene therapies.

Thank you once again to PPMD for an inspiring trip to Washington. We will use the knowledge gained to help our community here in the UK.  

If you are interested in learning more about patient advocacy and attending a training session with Duchenne UK, please email [email protected]


What is Duchenne Muscular Dystrophy? 
Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. 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. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300, 000 worldwide. It is classified as a rare disease.

Who are 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. 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. 

How to donate? 
Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via:

For more information and interview requests:
Visit www.duchenneuk.org
Molly Hunt – Communications Manager, Duchenne UK 
E: [email protected]

Published on 3 July 2019

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