Solid Biosciences announce preliminary results and plans to dose escalate for IGNITE DMD
Duchenne UK is delighted to share results from Solid Biosciences' gene therapy trial. Duchenne UK was an early supporter of this programme, and together with Joining Jack and the Duchenne Research Fund, committed $5million to support the programme.
Today Solid Biosciences announced interim results. In data published today the company said ….
This early data is encouraging and suggests the potential of gene therapy as a meaningful and important treatment for DMD.
Emily Crossley and Alex Johnson, co-founders/CEO’s of Duchenne UK, said:
We would like to thank the boys and their families for taking part in this trial and for the hard work & dedication of the team at Solid and the team at the University of Florida. This preliminary data is very encouraging and we are very hopeful that this will translate into a meaningful treatment for Duchenne. We are hopeful that all companies working in this space will continue to report positive data.
To read more about gene therapy click here. And to find out what else Duchenne UK is doing to address some of the challenges of gene therapy, please watch our film:
Two families a week in the UK are told that their sons have Duchenne Muscular Dystrophy, a devastating muscle wasting disease that mainly affects boys. We are an ambitious and highly focused charity, investing millions of pounds in research right now to bring treatments and a cure and to help this generation of boys. We rely entirely on donations. And we are in a race against time.
What is Duchenne Muscular Dystrophy?
Duchenne Muscular Dystrophy is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300,000 worldwide. It is classified as a rare disease.
Who are Duchenne UK?
Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne muscular dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children's Trust, the two biggest funders of DMD research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.
How to donate?
Duchenne UK is entirely reliant on donations to fund research for treatments and a cure to DMD. This can be done via: