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New 2018 Events New 2018 Events

12 January 2018 / Posted in: Help Us

Fancy a new challenge for 2018? Or holding a community event to raise vital SMA awareness? Check out our events!

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Fundraising Support Group Fundraising Support Group

12 January 2018 / Posted in: Help Us

Would you like to help SMA Support UK raise funds and awareness to ensure our vital services continue? If so, then why not consider joining our Midlands Fundraising Support Group!

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Transition to Adult Services

11 January 2018 / Posted in: Information & Support

Together for Short Lives has produced this factsheet to help parents think about the different elements of transition that need to be planned for and to provide tips to help the process feel as smooth as possible.

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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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Education, Health and Care Plans: Examples of Good Practice

02 January 2018 / Posted in: Information & Support

These resources have been produced to help practitioners write good quality EHC plans. The Council for Disabled Children suggests they may also be useful for parents, children and young people and those supporting them.

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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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AveXis Announce Phase 1 Trial of AVXS-101 for SMA Type 2

15 December 2017 / Posted in: Research

The Food and Drug Administration (FDA) has granted permission to AveXis to begin a second Phase 1 trial of their gene therapy drug, AVXS-101, with SMA Type 2 patients in the United States.

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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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New Phase 2 Study of Repurposed Drug for SMA

06 December 2017 / Posted in: Research

Catalyst Pharmaceuticals have announced that they are to investigate the potential of a drug called Firdapse as a treatment for people with SMA Type 3.

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