If you or your child live in England, have SMA Type 3 and have lost the ability to walk, please respond to this survey

20 November 2020

If NICE recommends next year that England's Managed Access Agreement for nusinersen should change to include you / your child, would you want to access this treatment?  

NICE has begun the process to review data collected as part of the Managed Access Agreement (MAA) for nusinersen. Specifically, it will consider whether people who have SMA Type 3 who are unable to walk can benefit from the treatment and therefore should be included in the MAA. NICE expects to publish a final decision in 6 months.

As it's important that everyone who might be eligible is contacted, SMA UK has agreed to put out this brief survey, via the Patient Groups, to help with any potential future planning. Asking for this information does not guarantee that a positive recommendation will be made.

Whether you are known or not to a Centre, please let us know if you would want nusinersen treatment. You may find it helpful to refer to information about the treatment, here.

Your response to this survey will help with planning but it does not commit you to any decisions or preferences you express – you will be able to change your mind. 

Please note your response to this survey is a personal opinion and not a referral for individual treatment. If the outcome of the review does recommend a change in the MAA, some eligibility /starting criteria will still be in place and you will need to be assessed by a medical team at one of the specialist neuromuscular treatment centres to discuss issues such as eligibility, your disease progression and administration.

It should take around 5 - 10 minutes to complete.

The closing date for this survey is Sunday 13th December 2020.

Survey for parents / guardians of children / young people aged 17 years and under

Survey for adults who have SMA and are aged 18 years and older