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, to help this generation of patients with DMD.
We believe strongly in partnerships, with academics and with biotechs. We fund all research, but we focus on translational projects, using our funds to help get promising therapies out of the laboratory and into the clinic where they can help patients. We belive in funding treatments that have a chance of making it to market, providing initial funding and then staying with them on that journey.
To understand our impact and strategy please take a look at our infographic: download or open here. And read on for more detailed information about the individual projects we have funded.
ReveraGen are recruiting for the clinical trial for Vamorolone which could offer a potential alternative to steroidsRead more
Our partnership with Solid Biosciences helped to advance their pre-clincial work on gene therapyRead more
A clinical trial for an anti-inflammatory drug that could reduce the thickening of the musclesRead more
Summit Therapeutics found new biomarkers for DMD that could lead to new drugs related to SMT C1100Read more
Professor Dickson explores the technique of gene editing - cutting out the mutations that cause DMDRead more
Duplications in DNA can cause DMD. We are funding gene therapy research that aims to correct this.Read more
Discovering new follow-on compounds to increase utrophin levels more effectively than SMT C1100Read more
This project looks at delivering a functioning full-size dystrophin gene via gene therapy transplicingRead more
A study to look at ways to improve stem cell transplants on a mouse model of DMDRead more
Professor Wood of Oxford University examines muscle tissue MicroRNAs to see if they are valid biomarkers for DMD.Read more
Stem cell and gene therapy combine in this new study which aims to repair muscle cells and tissueRead more
A study into the views of men with DMD about how to talk about end-of-life care and planningRead more
The project investigates how muscle fibre cells die in DMD and looks at ways to prevent thisRead more
We are funding a project to research a safe combination of existing drugs that could be used for DMDRead more
We are supporting work on CRISPR, a gene correcting tool that is showing promise as a treatment for DMDRead more
We fund a programme investigating if an exisiting compound can benefit DMDRead more
Our new research project aims to discover if and why a fermented soy product may help those with DMDRead more