Over 90 days have passed since NICE published their final guidance for Spinraza meaning that treatment should now be available for eligible patients with spinal muscular atrophy (SMA) in the UK.
SMA UK, TreatSMA and Muscular Dystrophy UK will all be representing the SMA Community as part of the Oversight Committee for the Managed Access Agreement (MAA). 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 Spinraza. An adult survey will follow in due course.
Whether or not you are interested in Spinraza 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.
You can take the survey here. The deadline for responses is 1 December.