For the first time in the history of this rare disease, the hope for treatments is becoming a reality. We are thrilled that our charity has been instrumental in driving this paradigm shift. The significant funds raised at our 2013 Gala Dinner have helped to launch the ground-breaking Solid GT gene therapy programme, support vital Duchenne clinicians and staff, and tackle the multi-faceted nature of this disease that impacts every muscle from legs and arms to heart and lungs.
But we urgently need to continue raising meaningful funds for three key reasons: firstly, we continue to pursue innovative research possibilities to fix every element of Duchenne. Secondly, we are focused on ensuring drugs are refined to be as impactful and safe as possible, as quickly as possible. And thirdly, we are collaborating with the NHS, charities and drug developers to ensure treatments are not delayed in getting out of the research labs and into clinical trials, to this generation of boys, who so desperately need them.
As we continually seek out the next generation of innovative research prospects, one particular area to keep an eye on is CRISPR – a cutting edge gene editing technology that is more precise, efficient and cheaper than its predecessors. Click here to read more about our Chief Scientific Advisor’s recent CRISPR breakthrough.