Page last updated: 29th January 2024

The Risdiplam Managed Access Agreement was agreed on 16th December 2021. The treatment programme started to roll out under the terms of the agreement on 17th January 2022:

• The Risdiplam Managed Access Agreement
• Who may access Risdiplam Treatment?
  • People who have been on Clinical Trials
  • Children who have had Zolgensma
  • Switching between Risdiplam and Nusinersen
• How can I access Risdiplam?
• Where are the Centres offering Risdiplam treatment, and other FAQs
• How long would it take to get my supply started at home?
• What data is being collected and why?
  • Clinical assessment scales and measures – what’s used, when?
  • Patient Reported Outcome Measures (PROMS)
• When might Risdiplam treatment stop?
• The Role of the NHS England SMA Clinical Panel
• The Role of the Managed Access Oversight Group

SMA UK and the other Patient Groups have all been part of the Managed Access Oversight Group​. 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.

Our 10th December 2021 webinar with a panel of experts discussed many questions raised by the Community about the treatment. On the panel were:

• Prof. Muntoni (Consultant Paediatric Neurologist, GOSH);
• Dr. Chiara Marini Bettolo (Consultant Paediatric and Adult Neurologist, Newcastle);
• Fiona Marley (Head of Highly Specialised Commissioning, NHS England & Improvement)