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. 
 
Patient Groups would like to find out what the views are of the SMA community and are putting out this survey for anyone who has SMA or is related to someone who has, or had, SMA.
  
SMA UK will collate all the surveys, 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.
 
Some questions about nusinersen (SpinrazaTM) are included as the submission form also asks what people think about current treatments and care.
 
For ease of reading, throughout this survey we refer to omnasemnogene abeparvovec by its trade name Zolgensma and to nusinersen by its trade name Spinraza. 
 
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.

If you haven't already done so, please open the accompanying Drug Treatments Summary Table as you'll need to refer to this as you respond.
 
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 could each person complete a separate survey.

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

Thank you.

T