Your Views on the Possible Provision of Zolgensma by NHS England, for Infants who have SMA Type 1

25 June 2019

The National Institute for Health and Care Excellence (NICE) is considering whether to recommend that NHS England funds the new drug treatment onasemnogene abeparvovec (also known as ZolgensmaTM)  for infants who have SMA Type 1. 

Although the survey asks only about access for infants who have SMA Type 1, the views of the wider SMA Community are valued and Patient Groups are putting out this survey for anyone who has SMA or is related to someone who has, or had, SMA.

As NICE wants to know what people think about current treatments and care, relevant information about ZolgensmaTM and SpinrazaTM has been summarised by members of Muscular Dystrophy UK’s research team and SMA UK’s Research Correspondent. This summary is for you to refer to as you complete the survey (you can find a link to this Drug Treatments Summary Table, below).

SMA UK will collate all the survey responses, summarise what we have been told and share the results with MDUK and TreatSMA. The anonymised statistical results of the survey may then be used to inform our Patient Group Submissions to NICE.

We would also like to be able to quote what people have told us. We may include non-identifying information to show the age / type of SMA that affects the person. You will be asked at the end of the survey whether you consent to this. Whatever your decision, SMA UK will not tell anyone your name or contact details. 

The survey should take around 20 minutes to complete, depending on how much detail you wish to include in your comments. You will need to complete it in one go as the survey doesn't allow for breaks. 

More than one person from any family is welcome to submit their views - please would each person complete a separate survey.

The closing date for this survey is 11.00pm Sunday 21st July 2019.

When you are ready to complete the survey, please open the accompanying Drug Treatments Summary Table  and then:

Complete the survey here

If you prefer a printed copy of the survey and / or summary table, or if you have any problems accessing or completing the survey, please contact or telephone 01789 267520.

Thank you