Felix's Fight Against Duchenne Muscular Dystrophy
August 09, 2023
Genomics and Therapeutics Development Research Fuels Hope and Treatments for Rare Disease
Ten year old Felix lives with Duchenne muscular dystrophy, a rare disease characterised by progressive muscle weakness. Because he has a gene mutation that can be treated, he is taking part in an early-phase clinical trial.
“The fact that we have another opportunity to feel hope – that feels good,” says Robyn, Felix’s mother. “It’s only good for the family if the drug works. We were discussing earlier…that for science, a fail is a win, because then they know what not to do next time.”
The University of Oxford is home to one of the largest concentrations of biomedical expertise in the world. The Oxford-Harrington Rare Disease Centre (OHC) is housed at the Institute of Developmental and Regenerative Medicine, part of the University’s biomedical research campus. It’s through partnerships – nationally and internationally – that the University is committed to using its expertise in research in genomics and therapeutic development to make a real difference in the lives of those living with rare diseases.
Finding a genetic diagnosis and identifying novel treatments for thousands of rare diseases continues to be the goal. Working with the OHC, global leaders aim to progress 20 new treatments into clinical trials over the next ten years.
“We hope that each rare disease therapeutic we can accelerate will impact more than one disease so that we amplify the power of science, innovation, and our impact on patients and their families living with rare disease,” said Professor Matthew Wood, Director of the OHC.
For Rare Disease Day 2023, the Oxford-Harrington Rare Disease Centre (OHC) welcomed ITN Business and Genetic Alliance UK to make a short film following Felix, a ten year old boy living with the rare, genetic, neuromuscular condition Duchenne muscular dystrophy. The film follows Felix on one of his visits to Oxford, and describes how the OHC and its partners are working together to harness expertise in genomics and therapeutic development to deliver treatments for children suffering with rare, genetic diseases. Please watch the video to learn more and to hear OHC Director Prof Matthew Wood outlining his vision for the Centre.