“THERE ARE NO MIRACLES - THERE’S JUST UNDISCOVERED SCIENCE”

This week, Alex and I will be heading to Copenhagen for the World Muscle Society meeting, a gathering of scientists, clinicians, researchers, funders and pharmaceutical companies as well as many other people all working in muscle disease.

For Alex and I, travelling for work is always challenging. We struggle being away from our sons and our families and find it hard to keep our heads together and put on a brave face for days at a time, when inside we are grieving. But I read a quote last week which reminds me why we keep going to these and other meetings. The reason why we keep our heads down and why we are constantly searching, constantly looking and constantly questioning what we need to fund and where we need to look next is because finding treatments for Duchenne Muscular Dystrophy is not easy – and it’s because there are no miracles, there’s just undiscovered science.

We’ve uncovered some great projects at previous meetings.

In 2016, our then research director, Fiona Lawrence, heard about early work done by Prof Dirk Fischer on tamoxifen, and met up with him. Alex and I then went with Fiona to meet him in Switzerland, and 3 years and a £1.5million investment from Duchenne UK later, the trial is running in Europe and the UK with recruitment active now and more than 40 patients have been dosed.

Our current research Director, Dr David Bull, will be joining us and it was at WMS last year that he found out about work on the anti-oxidant Tempol, a project that we are funding and is now underway.

This year we will be raising our profile and telling delegates about what Duchenne UK is doing, and we will be shouting loudly about getting the patient voice heard! It’s why your feedback to us is so important, because it’s at meetings like this that we can represent your views.

We’re proud to be partnering with Parent Project Muscular Dystrophy (PPMD) in the joint presentation of our work on the parent voice at a stand in the main conference hall. This is the first time we’ve had a presence in this way and is an opportunity to explain how we have helped to change the treatment landscape in Duchenne; how we are partnering with PPMD in the funding of new research, and help researchers to understand the impact organisations like DUK and PPMD have had on policies and drug development.

We are at WMS on your behalf – the Duchenne UK team, with the support of our parents, carers and boys, have done amazing things in the last few years but we cannot rest yet. There is more for us to do and the WMS conference may give us our next lead or introduce us to an organisation we can partner with to move faster.

So, greetings from Copenhagen as we continue our search for the undiscovered science and the clinical trials that we want to fund more of. 

Thank you for your support.

Emily & Alex

Co-Founders Duchenne UK

http://www.wms2019.com

 

 


NOTES FOR EDITORS

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy (DMD) 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.

We are investing millions of pounds in research right now to bring treatments and a cure to help this generation. Duchenne UK is the largest funder of DMD research in the UK. We are also committed to accelerating the pace of research. 90p in every £1 raised is committed to research.

Our president is HRH The Duchess of Cornwall. Our patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell. 

We need your help, because we need to keep funding promising new research.

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:

  • Direct Debit – Duchenne Direct
  • Individual Donation – Donate
  • If you are a family or friend affected by DMD you can set up your own fund with Duchenne UK – Family and Friend Funds
  • Take part in one of our fundraising events – Events
  • Text DUCHENNE to 70085 to donate £5. This costs £5 plus a std rate msg.

For more information visit www.duchenneuk.org