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how you can help

Last updated 19th January 2018.

We are working with Muscular Dystrophy UK, the SMA Trust, and the family-led campaign group TreatSMA. Your letters and lobbying of MPs, NHS England and NICE have helped immensely to get the SMA Type 1 EAP funded by NHSE in the UK. Thank you.

If you live in England

Yesterday, the National Institute for Health and Care Excellence (NICE) started its appraisal of nusinersen for the treatment of all types of SMA. It is now looking at the treatment via the Single Technology Appraisal (STA) route. This 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

We have been invited to make a Patient Group Submission by 5th February 2018. Thank you to those of you who completed our survey to help us prepare for this. We will post the submission on our website when it is ready.

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.

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