What We Do News We Fund New Therapeutic Approach Duchenne UK is pleased to announce a collaboration between EspeRare, a not-for-profit drug development organisation, and Professor Dominic Wells at the Royal Veterinary College, to test a potential new anti-inflammatory and anti-fibrotic therapy on the mdx mouse model for Duchenne. Those living with Duchenne lack the muscle protein dystrophin which acts as a ‘shock absorber’ for muscle. Without a shock absorber, everyday use of muscles cause them to become damaged. This damage promotes inflammatory processes which in turn cause hardening and scar formation. Muscles damaged in this way are termed fibrotic. It is hoped that this potential new therapy will be effective in treating both inflammation and fibrosis in the damaged muscles of those living with Duchenne. Duchenne UK is providing £67,980 to fund an eight-month programme looking at an existing compound to see if it will have benefit in DMD. The preclinical studies we are funding will look at the scientific rationale to “re-position” the compound as a treatment for DMD. This would be a treatment for all those living with Duchenne, regardless of their age or DMD gene mutation. This compound has already been tested in several animal models of inflammation and fibrosis. It has also been tested for safety in healthy volunteers, in several Phase I studies as well as in a Phase II study. If this research programme shows promise, the compound will be able to go straight to a Phase II study in DMD patients. In line with our commitment to bring medicines to market, Duchenne UK commits to help fund a Phase II study, if the compound shows efficacy. Emily Crossley and Alex Johnson, co-founders of Duchenne UK, said: We are delighted to be able to join forces with EspeRare and the Royal Veterinary College, to confirm the efficacy in DMD and advance our knowledge about this compound. By leveraging on the available data package and capitalising on its safe use in humans in previous studies, we hope that, if it shows promise, we could go into patients with DMD as soon as early 2018. Florence Porte, CSO of EspeRare said: Thanks to the support from Duchenne UK, it has been possible to transform this shelved asset into a potential novel therapeutic option for patients with DMD. This innovative repositioning approach may offer to patients with DMD a new and safe disease modifier addressing inflammation and fibrosis and which may be combined with dystrophin replacing therapies.