Authors' objectives: Spinal muscular atrophy is a long-term condition that causes muscle weakness. The weakness gets worse over time and results in a loss of movement. Spinal muscular atrophy is a genetic condition that is caused by a fault (or mutation) in a gene called the survival motor neuron gene 1. It is inherited in a way that means both parents must be carriers of the disease for the offspring to be affected. Nusinersen is injected into the spinal cord of infants and children with early spinal muscular atrophy. There is currently no cure for spinal muscular atrophy, but medical interventions can improve symptoms. Nusinersen is currently being studied to see how well it works and whether it is safe to use in people with spinal muscular atrophy. If nusinersen is licensed in the UK, it will offer the first treatment specifically for people with spinal muscular atrophy.

Project Status: Completed

Year Published: 2016

URL for published report: http://www.hsric.nihr.ac.uk/topics/nusinersen-for-paediatric-onset-spinal-muscular-atrophy-first-line/

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)