Spinraza J.Kingsley@mus… Thu, 04/01/2021 - 12:48

It is currently available to eligible people with SMA through a Managed Access Agreement (MAA) in England (which also covers Wales and Northern Ireland) and MDUK is a member of the Managed Access Oversight Committee (MAOC). It is available in Scotland through the ultra-orphan pathway.

In May 2021 NICE extended the clinical eligibility criteria of the MAA to allow access to nusinersen for those with SMA Type 3, who aren’t able to walk. The review also removed the rule that meant those who lost the ability to walk after 12 months of treatment would no longer be eligible for further treatment.

Information and guidance

NICE, NHS England and NHS Improvement have taken advice from clinical experts and consulted with patient advisory groups Muscular Dystrophy UK, Treat SMA, and SMA UK to produce a series of question-and-answer documents relating to Spinraza.

Access for Children Q&A (PDF, 407KB)
Managed Access Agreement Q&A (PDF, 425KB)
Access for Adults Q&A (PDF, 155KB)
About Spinraza (Word, 21KB)
Treatment process (Word, 21KB)
Spinraza data collection (Word, 24KB)