Authors' objectives: Duchenne muscular dystrophy (DMD) is a life-limiting condition that mainly affects boys. There are around 2,500 people in the UK living with the condition. It is an inherited disease caused by a fault (or mutation) on a gene called the dystrophin gene, which is very important for healthy muscles. Parents of children with Duchenne muscular dystrophy often begin to notice the signs of muscle weakness (such as difficulties in getting up off the floor) when their children are aged 2-3 years old. In boys with Duchenne muscular dystrophy, muscle weakness slowly gets worse and most patients will need to use a wheelchair by the time they are 10-12 years old. There is no cure for Duchenne muscular dystrophy and most drug treatments that are available for patients now, aim to stop the muscles getting weaker and allow patients to walk for as long as possible, but they are not fully effective and can have unpleasant side effects. Tadalafil is a new drug for the treatment of Duchenne muscular dystrophy that is given once a day as a tablet. Some studies have suggested tadalafil may be helpful for boys with Duchenne muscular dystrophy who are still able to walk and more studies are now aiming to show how well it works and that it is safe to use. If tadalafil is licenced for use in the UK, it could be a new treatment option for boys with Duchenne muscular dystrophy who are still able to walk. Tadalafil may slow the rate of muscle decline and allow patients to walk for longer.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/tadalafil-cialis-for-duchenne-muscular-dystrophy/

English language abstract: An English language summary is available

Publication Type: Not Assigned

Country: England, United Kingdom

Organisation Name: NIHR Horizon Scanning Centre

Contact Address: The NIHR Horizon Scanning Centre, Department of Public Health, Epidemiology, and Biostatistics, School of Health and Population Sciences, University of Birmingham, 90 Vincent Drive, Edgbaston, Birmingham, B15 2SP. United Kingdom. Tel: +44 121 414 7831, Fax: +44 121 2269

Contact Name: c.packer@bham.ac.uk

Contact Email: c.packer@bham.ac.uk

Copyright: NIHR Horizon Scanning Centre (NIHR HSC)