Page last updated: 29th January 2024

The Nusinersen Managed Access Agreement was first agreed in July 2019 and, following review, has since been updated. Our website pages summarise what’s happening now:

• The Nusinersen Managed Access Agreement
• Who may access Nusinersen Treatment?
  • People who have been on Clinical Trials
  • Children who have had Zolgensma
  • Switching between nusinersen and risdiplam
• How can I access Nusinersen?
• Where are the Centres offering Nusinersen treatment, and other FAQs?
• Access for children – other FAQs
• Access for adults – other FAQs
• What data is being collected and why?
  • Clinical Assessments scales and measures – what’s used, when?
  • Patient Reported Outcome Measures (PROMS) via the UK SMA Patient Registry
• When might nusinersen treatment stop?
• The Role of the NHS England Clinical Panel
• The Role of the Managed Access Oversight Committee

SMA UK and the other Patient Groups have all been part of the Managed Access Oversight Committee. If you are concerned about your access, please talk first and foremost to your medical team, but do please get in touch if you want to discuss your access.