Our vision is to witness a world without Duchenne Muscular Dystrophy
We want a world where Duchenne becomes a manageable and treatable condition that can be lived with. We want a future where the devastating impact Duchenne has on our children no longer exists. A future where they can grow and prosper and fulfill their dreams whilst not living trapped in their own bodies. A life where they can enjoy every single day without the fear of what Duchenne will bring.
Our vision is to cure everyone affected by Duchenne and to bring new treatments to this generation of children and young adults.
Everything we do is with this in mind.
Within the next 10 years, we want to stop the devastating effects this condition has on boys and young adults with Duchenne. We want to save their lives.
We will achieve this by working closely with Duchenne UK and the leading scientists from around the world to bring potential viable new life-saving drugs to market.
- We are a small but growing charity with a big heart
- We go the extra mile in making our supporters feel valued
- We are positive, upbeat and committed totally to our aim
- We are totally transparent
Our urgent need for funding
We need to provide substantial funding to several clinical trial/research projects as we well as fund vital clinical posts to run these trials over the next 3 to 5 years. This will provide a catalyst to drive significant investment from biotech and pharmaceutical companies. We have a target of £1,000,000 raised by end 2020.
A significant factor in getting treatments out of the laboratory and into clinic is funding! Rare diseases get very little funding from the government so it is down to charities such as Alex’s Wish to do it. It is likely multiple drugs and treatments will be needed to repair, restore or replace the missing protein in Duchenne called ‘dystrophin’. This missing protein is what causes the disease.
Having met and spoken to some of the best organizations in the world fighting Duchenne it is clear that for the very first time there is likely to be a chance of a major breakthrough. This generation of children could be the ones to survive this condition – but only if action is taken now.
With only one treatment available Translarna which was given the green light by NICE in April 2016 will only treat around 13% of the Duchenne population, that’s 50 boys in UK, we have to work hard to find more treatments that will treat ALL boys and not just a sub-set. However, with the first drug that treats the underlining effects of Duchenne, it is a huge step in the right direction.