We want to eradicate Duchenne Muscular Dystrophy by raising funds and supporting research/clinical trial projects and support clinical trial capacity to ensure that there are enough staff in place to run the increasing number of trials taking place.
We are a charity partner to Duchenne UK working with them towards a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy.
We will only invest in projects that are sharply focused on advancing research and getting treatments into clinic and to patients as quickly as possible. We will only invest in projects that have a clear route to market and have a chance of becoming available to help this generation of children. We will only invest in projects that have the opportunity of helping all children/young adults, rather than just a sub set of individuals.
We want a future where the devastating impact of 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 day without fear of what Duchenne will bring.
We have set ourselves a target to raise £1,000,000 by 2020. When we have achieved this we will continue as a charity to raise money until Duchenne has been eradicated.
The effect of Alex’s Wish achieving this ambition is extremely important. For the first time, there is likely to be a chance of a major breakthrough in the treatment and cure of Duchenne Muscular Dystrophy. This generation of children could be the ones to survive this condition – but only if action is taken now and that means fundraising to ensure that clinical research, clinical trials and clinical trial capacity is maximised and all viable opportunities are funded.