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. Tadalafil (Cialis) for Duchenne muscular dystrophy. Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2015 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. Indexing Status Subject indexing assigned by CRD MeSH Humans; Muscular Dystrophy, Duchenne; Tadalafil 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 32016000390 Date abstract record published 04/03/2016 |