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.
 
NHS England and the Neuromuscular Centres need to be ready for the possibility of a positive recommendation for change. 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, even though a positive recommendation may not be made. Whether your child is known or not to a Centre, please let us know if you would want them to have nusinersen treatment so that we can pass this information on to help with service planning.
 
Please note your response to this survey is not a referral for individual treatment, which must be via a medical team. If the outcome of the review does recommend a change in the MAA, other eligibility /starting criteria will still be in place and your child will need to be assessed by a medical team at one of the specialist neuromuscular treatment centres.
 
Your response to this survey will help link you and your child with NHS England and the Neuromuscular Centre(s) that would provide this service if it is recommended, but it does not commit you to any decisions or preferences you express – you will be able to change your mind.
 
SMA UK will collate all responses to this survey and share the information with NHS England and the Neuromuscular Centres.
 
You will be asked at the end of the survey what information you consent to being shared.

It should take around 5 - 10 minutes to complete.

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

Thank you.

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* 1. Please tell us who is completing this survey

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* 2. Please confirm that your child lives in England before completing the survey

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* 3. How old is your child with SMA Type 3?

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* 4. Please give the first half of your child's postcode (letters/digits).

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* 5. Does your child have a scoliosis / spinal intervention (had or planned) that may prevent a clinician from being able to safely administer nusinersen treatment via lumber puncture?

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* 6. The Centres listed below currently have a contract that allows them to offer treatment with nusinersen. Please tick any ones which provide your child with care and management for their SMA. 

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* 7. The Centres listed below currently have a contract that allows them to offer treatment with nusinersen. Please tick the one that is nearest to you and, if different, the one you would like your child to attend if they were eligible for treatment.

  Nearest Preferred
Alder Hey Children’s NHS Foundation Trust
Cambridge University Hospitals NHS Foundation Trust
Great Ormond Street Hospital for Children NHS Foundation Trust
Guy’s & St Thomas’ NHS Foundation Trust
Leeds Teaching Hospitals NHS Trust
Manchester University NHS Foundation Trust
Nottingham University Hospitals NHS Trust
Oxford University Hospitals NHS Foundation Trust
Sheffield Children’s NHS Foundation Trust
The Newcastle upon Tyne Hospitals NHS Foundation Trust
University Hospital Southampton NHS Foundation Trust
University Hospitals Bristol NHS Foundation Trust
University Hospitals of North Midlands NHS Trust
I do not have care and management from any of these Centres

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* 8. Do you have  your own transport and support to get your child safely to and from appointments at the treatment centre(s) you have selected?

  My nearest Centre My preferred Centre
Yes
No 
Don't know 

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* 9. If you said 'no' or 'don't know' to Q.8, what transport and /or support would you need to get your child safely to and from treatment appointments?

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* 10. Is there anything else you would like to tell us?

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* 11. Please let us know if you are willing to share your name and contact details with NHS England and the treatment centre(s). This will be a huge help for planning purposes. We will not share this information with anyone else.

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* 12. If you would like to tell us who you are, please complete the details below. We will make sure to follow the instruction you have given us in your answer above.

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