Authors' objectives: Duchenne muscular dystrophy is an inherited condition that mainly affects boys and causes muscle weakness. Most people with Duchenne muscular dystrophy are diagnosed by the age of 5 years, and need to use a wheelchair by the age of 12 years. Many will face severe health problems by their late teens as their heart muscle and chest muscles become weaker, eventually affecting their breathing. Idebenone is a new drug for the treatment of Duchenne muscular dystrophy given as a tablet three times a day. It is being studied to see how well it slows down the weakening of breathing and heart muscles, and to see if it is safe for patients to take. If idebenone is licensed for use in the UK, it could be a new treatment option for patients with Duchenne muscular dystrophy and may reduce symptoms of the disease and improve survival. At the moment, there are no drugs that treat the underlying cause of Duchenne muscular dystrophy.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/idebenone-raxone-for-duchenne-muscular-dystrophy-first-line/

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 Research&Intelligence Centre (NIHR HSRIC)