DRF Patron Evening
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Recent news
Catch up on the latest developments in the projects we fund.
FDA approves first gene therapy for Duchenne
The FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world …
DRF highlights psychosocial research at parent information day
The DRF was pleased to participate in Duchenne UK’s latest parent information day, held in March in central London.
See the smart suit that is changing children’s lives
On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart suit’ aims to help children who are losing upper body function in still continuing to use their arms.
Our mission
We aim to stop Duchenne muscular dystrophy being a death sentence for hundreds of thousands of boys worldwide.
Our impact
We are helping turn the hope of treatments into reality. Critical supportive drugs are in clinical trial thanks to our funds.
Your help
In the last 3 years we have given more than £1.5m to ground-breaking research projects.
And there is still work to do.