SMA UK Sends Evidence to Biogen for the NICE Review Considering Whether People who have SMA Type 3 and Unable to Walk can Benefit from Nusinersen Treatment

23 November 2020

We are very aware that this review will be focusing on clinical evidence from Biogen and the clinical community. As part of this, we consider it is vital that NICE hears Patient Reported Outcomes. Thanks to the families in the UK and adults in Europe who have allowed us to pass on their reports. We have also submitted evidence of the impact the decision to deny access has had on those who have lost the ability to walk – our thanks to those who have given us permission to pass on their comments. And, once again, we have challenged the thinking that led NICE to make their original decision.

Submission summary

Adults treated in Europe

The Impact of not being eligible to access treatment - survey results