Research and trials

research

Experts truly believe for the first time in history that a major breakthrough is likely in the next 5-10 years.

Over the past 2 years, scientists has made significant advances in molecular medicine, gene therapy and combination treatments. Professor Dame Kaye Davies quoted earlier this year that ‘at least 1 effective treatment will be available in the next 5 years’. So huge progress is being made. Click here to listen to this interesting video about Duchenne research.

Alex’s Wish is part of a network of foundations and scientists dedicated to conquering Duchenne.  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.

GLOBALLY

Duchenne Alliance

 

 

Alex’s Wish is an active member of the Duchenne Alliance – an alliance of 40+ independent international Duchenne organisations. Together we have taken ‘the ultimate race against time’ boys with Duchenne don’t have any time to waste – we need action and and we treatments right now. To date, we’ve pledged £230k into promising trials on the dashboard, and we’re looking into more promising research right now.

IN THE UK

 

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

AMETEK

A huge thank you to American giant AMETEK who donated $30K to Alex’s Wish in late 2013 for two of the projects listed. AMETEK Inc. is a leading global manufacturer of electronic instruments and electromechanical devices with annual sales of $3.6 billion. One of their colleagues from the UK, Nick Maddock (his daughter is in the same class as Alex’s) put Alex’s Wish forward.