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. Cerliponase alfa for neuronal ceroid lipofuscinosis type 2 (CLN2 disease) Birmingham: NIHR Horizon Scanning Research&Intelligence Centre. Horizon Scanning Review. 2015 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. Indexing Status Subject indexing assigned by CRD MeSH Dipeptidyl-Peptidases and Tripeptidyl-Peptidases; Humans; Neuronal Ceroid-Lipofuscinoses; Serine Proteases 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 32016000415 Date abstract record published 04/03/2016 |