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Scottish Medicines Consortium Assessment

Last updated 2nd March 2018

Thank you to those of you who responded to our invitation to take part in a survey to tell us about your experiences of SMA and your views on access to the treatment. We shared a summary of what you had said with the charities Muscular Dystrophy UK and the SMA Trust and with TreatSMA - the campaign group. We made a Patient Group Submission (PGS) to the SMC, as did MDUK and the SMA Trust. You can read a copy of our submission here and a summary of the survey results here.

On 15th February, the Scottish Medicines Consortium (SMC) advised that, ‘No economic case for use of nusinersen in pre-symptomatic patients, “those who are most likely to develop SMA type I, II or III” was made to SMC.'

They have now announced that there will be a Patient and Clinical Experts (PACE) meeting on March 13th 2018 when they will consider the submissions and evidence provided by groups ‘for use in patients with infantile‑onset SMA (those who have or are most likely to develop SMA type I), and in patients with later‑onset SMA (those who have or who are most likely to develop SMA type II or III).’ Our representatives, Kathy Huffman, bereaved mum of Maggie who had SMA Type 1 and was unable to access treatment and Liz Ryburn, Support Services Manager, will join MDUK and the SMA Trust to make a joint presentation. The meeting is not open and what is discussed has to remain confidential at this stage.

We will let you know as soon as soon as we have any further updates from the SMC that we can share publicly.