Authors' objectives: Neuronal ceroid lipofuscinosis (or CLN2 disease) is a very rare inherited disease that affects young children. The disease affects brain cells, and leads to blindness, fits (epilepsy), difficulties with movements and learning difficulties. At the moment, there is no cure, and even with current treatments, children usually die by the age of 12 years old. Cerliponase alfa is a new drug being developed to treat neuronal ceroid lipofuscinosis. It has to be given in hospital every two weeks. A study is currently looking to see whether cerliponase alfa helps to slow down the disease and is safe for to take. If it is licensed for use in the UK, cerliponase alfa will be the first drug available to treat this rare childhood disease.

Project Status: Completed

Year Published: 2015

URL for published report: http://www.hsric.nihr.ac.uk/topics/cerliponase-alfa-for-neuronal-ceroid-lipofuscinosis-type-2-cln2-disease/

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)