Authors' recommendations: GNE myopathy is a very rare inherited disease that affects young adults. The disease affects muscles in the legs, making it difficult to walk and get up from sitting. Muscles weaken and symptoms get worse slowly over time, with the disease eventually affecting the arm muscles too. At the moment, there is no cure and patients eventually end up in a wheelchair. Ace-ER is a new drug being developed to treat GNE myopathy. It is given as four tablets taken with food three times a day. A study is currently looking to see if Ace-ER can slow down the disease and that it is safe to take. If it is licensed for use in the UK, Ace-ER will be the first drug available to treat this rare inherited disease.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/ace-er-aceneuramic-acid-extended-release-for-gne-myopathy/

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)