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how you can help

Last updated 21st February 2018.

We are working with Muscular Dystrophy UK, the SMA Trust, and the family-led campaign group TreatSMA. 

If you live in England:

  • Thank you for responding to our surveys. These will inform our submissions.
  • ‚ÄčTreatSMA is collecting experiences for their submission and would like to hear from families affected by SMA Type 1 Read more
  • Talk to the media. If you are willing to do so, contact MDUK. Read more
  • Write to your MP:

The STA is designed to assess drugs for both clinical- and cost-effectiveness and is usually used for more common conditions. As such, we are concerned that this route is not set up to assess rare disease drugs, like nusinersen.  

However, there is some promising news. Biogen, has announced that they are in discussions with NICE and NHS England to put an interim access scheme in place. This scheme, known as a Managed Access Agreement (MAA), could give others with SMA access to the treatment while further data and evidence are gathered over several years.

Time is key and it is crucial nusinersen is not subjected to any delays during the NICE assessment process. We have worked with MDUK and the other charities to prepare a template letter that you can use to write to your MP You can download the letter here and find your MP’s address here. Please do send us a copy if you do write.

If you live in Scotland

Thank you to those of you who completed our surveys to help us prepare for our submission. 

Wherever you live in the UK

You can back MDUK’s ‘Fasttrack' campaign which includes access to nusinersen (Spinraza) by using their template letters to write to your MP and signing their petition asking for the Prime Minister’s help in removing barriers to all life-changing treatments that people would benefit from.

To keep up to date with progress and developments you can follow us on Facebook and Twitter and sign up to receive our monthly E-news updates.