Action Duchenne is the only UK charity exclusively fighting for a cure for Duchenne. They have done some really incredible work raising funds and awareness in the UK and worldwide. They work grass roots all the way to the government. Their mission is similar to Charley’s Fund in that they focus heavily on funding research to find effective therapies for DMD. They have also supported research at AVI BioPharma. Their board of Directors are all parents or grandparents of people living with Duchenne. One of their patrons is Helena Bonham Carter. She is a talented actress and mother of two, and she recently spoke out to urge the government in the UK to adopt certain practices that would better equip their National Health Service in serving patients who require treatment with genetic medicines in the near future. A recent press release by PR Artistry outlined some of the concerns and also how Action Duchenne is pressing the government to adopt best practices in the way that NHS administers care to DMD patients.
Bonham Carter’s direct quote is “Recent developments in genomic medicine are very exciting, but are still thwarted by lack of funds. It is incredibly important that single-gene disorders like Duchenne Muscular Dystrophy are not left behind in the rush to treat more widespread diseases like cancer or diabetes. Scientists are very close to significant breakthroughs with treatments such as exon skipping, and it is vital that they received adequate funding and support from the Government, rather than relying on charities to provide funds. We also need to make sure that the health service is able to deliver treatments for boys and young men with Duchenne, as soon as they are developed.”
CEO of Action Duchenne, Nick Catlin, added “The recognition that genomic medicine is a significant step forward is very exciting, as Duchenne is a disorder that looks very likely to benefit. However, we are very concerned that single-gene disorders like Duchenne Muscular Dystrophy that are relatively rare will now become the poor relation as funds are diverted to more complex conditions. This must never be allowed to happen. We would argue that Government should concentrate on building a delivery model for genomic medicine for single-gene diseases, like Duchenne, where the cause is already identified, and therefore easier to deal with. This should be based on ring-fenced funding for a programme of research and clinical trials, and roll out of Centres of Excellence where working with experts means that more clinicians become trained in the delivery and monitoring of these new genomic treatments and drugs.”
These are just more examples of people who are working very hard to make sure that research is funded and awareness is raised. The sense of urgency among the DMD community is really inspiring. The science, the outreach, the hope and the possibilities are all extremely exciting. We all have an opportunity to make a difference and see a breakthrough therapy for this heartbreaking disease. Donate to Charley’s Fund, buy a Darius Goes West DVD, buy the Live Inspired app for the iPhone. Now is the time.