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Latest News: Research

Opportunity to let NICE hear your views about the impact of 5q SMA and who should be able to access nusinersen

12 February 2018 / Posted in: Research

SMA Support UK has set up a number of surveys so that we can collect different people’s views and share them with MDUK, SMA Trust, TreatSMA, and NICE. Deadline to complete survey(s) is 18th Feb.

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Our Patient Group Submission to the Scottish Medicines Consortium (SMC)

06 February 2018 / Posted in: Research

Thanks to those 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 nusinersen. Read a copy of our submission and a summary of the survey results, here.

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Rare Disease Implementation Plans for England Published

01 February 2018 / Posted in: Information & Support, Research

The Department of Health and Social Care and NHS England have published their implementation plans for the UK Strategy for Rare Diseases. England is now in line with Scotland, Wales and Northern Ireland who already have plans in place.

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AveXis Announce Additional Trials of AVXS-101 for SMA

25 January 2018 / Posted in: Research

AveXis have provided an overview of their expanded program of clinical development for their gene therapy drug AVXS-101 in SMA, which incorporates two new trials to include a broader set of SMA patients.

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Biogen’s SMA Community Update Confirms the NICE Appraisal Process

22 January 2018 / Posted in: Research

It also contains a little bit more information about how the possibility of a Managed Access Programme will be explored.

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NICE Announces Appraisal Route for Nusinersen

19 January 2018 / Posted in: Research

This will be via the Single Technology Appraisal (STA) route which is not set up to assess rare disease drugs. However, there is some promising news which may see an interim Managed Access Agreement (MAA) put in place which could give more people with SMA access.

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The Updated Standards of Care Have Now Been Published

08 January 2018 / Posted in: Research

This means work can now start on writing the updated Family Guide. SMA Support UK will be working with others on this. In the meantime, you can read a summary of the ENMC workshop review of the standards.

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Invitation to parents/main carers in the UK whose children have received nuisnersen treatment to voice your views

19 December 2017 / Posted in: Information & Support, Research

Wherever you live in the UK, we would like to be able to include your views in our Patient Group Submission to the Scottish Medicines Consortium.

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Invitation to the Scottish SMA Community to voice your views

19 December 2017 / Posted in: Information & Support, Research

The Scottish Medicines Consortium (SMC) will shortly be assessing the treatment nusinersen (Spinraza) to decide whether it can be used by NHS Scotland for the treatment of SMA Types 1, 2 and 3. We need your help with this.

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Summary and comparison of results to date for nusinersen and gene therapy (Avexis-101)

15 December 2017 / Posted in: Research

Dr Alex Murphy, our Clinical Care Research Correspondent, reviews two recent articles published in the November New England Journal of Medicine.

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