Survey About What Progress There Has Been With Children's Access To Nusinersen In The UK

14 November 2019

Over 90 days have passed since NICE published their final guidance for nusinersen meaning that treatment should now be available. SMA UK, TreatSMA and MDUK will all be representing the SMA Community as part of the Oversight Committee for the Managed Access Agreement. The first meeting is not until next year but we remain active. We are currently seeking updates from NHS England and Biogen regarding which centres will be providing treatment for paediatric and adult patients so we can keep the Community informed. We also want to hear about the experiences of the SMA Community.

Today, we have launched a survey for the paediatric SMA Community to gather information about people’s experiences of trying to access nusinersen (SpinrazaTM). An adult survey will follow in due course.

Whether or not you are interested in nusinersen treatment for your child and whether or not they are currently being treated or waiting for access, if you live in the UK and you are the parent or guardian of a child / young person (aged 0 – 18 years) who has been diagnosed with 5q SMA (Types 1, 1/2, 2, 2/3 or 3) we'd like to hear from you. By completing our survey you’ll be contributing to the wider understanding of where access is now:

Please complete our survey here

Closing date: Sunday 1st December 2019