What We Do Funding Research & Trials Montelukast: Repurposing of an anti-inflammatory drug to potentially promote muscle strength and movement February 2017 Duchenne UK is investing $180,000 into research into the use of the anti-inflammatory drug montelukast, as a potential therapy for Duchenne Muscular Dystrophy. Drs. Dongsheng Duan and Robert McDonald from the University of Missouri will lead the study. Duchenne UK is pleased to be partnering with Ryan’s Quest, Michael’s Cause and Rally for Ryan, all US based charities, who are each contributing $50,000 to the project. This research will look in to how safe and effective montelukast is, as a standalone therapy for Duchenne muscular dystrophy. It will also look at the drug in combination with corticosteroids. This research will be conducted in mouse models. Montelukast is a type of medicine known as a leukotriene receptor antagonist. It works by blocking the action of leukotrienes which are chemicals released by the body as part of inflammatory reactions. Montelukast (brand name Singulair) is licensed as a treatment for asthma. Duchenne UK is interested in exploring the potential of montelukast as a treatment for DMD. Our ultimate goal is to determine if montelukast is safe and effective as a lifelong therapy for DMD. Notes for editors For more information, please visit our website at www.duchenneuk.org For interview requests contact Molly Hunt: [email protected] Further notes: 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 by the disease, 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 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 Kris Radlinski and Andy Farrell.