Record Status This is a bibliographic record of a published health technology assessment. No evaluation of the quality of this assessment has been made for the HTA database. Citation NIHR HSRIC. Idebenone (Raxone) for Duchenne muscular dystrophy – first line. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2016 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. Indexing Status Subject indexing assigned by CRD MeSH Humans; Muscular Dystrophy, Duchenne; Ubiquinone Language Published English Country of organisation England English summary An English language summary is available. Address for correspondence NIHR Horizon Scanning Research&Intelligence Centre, University of Birmingham, Institute of Applied Health Research, Public Health building, Edgbaston, Birmingham B15 2TT
Tel: 0121 414 9077 Email: nihrhsc@contacts.bham.ac.uk AccessionNumber 32016000439 Date abstract record published 08/03/2016 |