Experts truly believe for the first time in history that a major breakthrough is likely in the next 5-10 years.
Over recent years scientists has made significant advances in molecular medicine, gene therapy and combination treatments. Professor Dame Kaye Davies quoted recently that ‘at least 1 effective treatment will be available in the next 5 years’. In the short space since Alex’s diagnosis back in 2010, we have seen so many clinical trials now taking place – with over 60 across the world. There is more hope now than there has ever been, but we cannot stop the vital work we do – without ongoing funding new treatments will not become available.
We only invest in research that has been through a scientific advisory board. Our aim is only to invest in the most promising research that has the best chance of curing or treating this generation of children.
Alex’s Wish is an active member of the Duchenne Forum a collaboration established between the Muscular Dystrophy UK and 5 other charities. Together we accelerate progress in the search for effective treatments and eventually cures. This partnership is supporting seven pioneering Duchenne research projects, to which it will commit £840k over the next 4 years. The seven projects include:
- Up-regulation of utrophin
- Developing small molecules to help increase utrophin levels
- Gene therapy
- Genome surgery – this ground-breaking technique could have the potential to be the first therapy to offer a permanent correction of the genetic mutation in a person’s own DNA
- Investigations into inflammation which is known to contribute to muscle-wasting
- Stem cell treatment
- Identification of biomarkers – to help improve diagnosis, measure disease progression and assess benefit of potential drugs in clinical trials