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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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Big Success in the Big Give 2017! Big Success in the Big Give 2017!

19 December 2017 / Posted in: Help Us

We were overwhelmed to receive so many donations - you helped us to exceed our target and reach over £13,000!

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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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Update on the Scottish Medicines Consortium's Assessment of Nusinersen

30 November 2017 / Posted in: Research

We have now received a further update and clarification that the Scottish Medicines Consortium will be assessing nusinersen for "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA".

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Petition: Supermarkets To Commit To Providing Changing Places Toilets In Stores Within 18 Months

29 November 2017 / Posted in: Information & Support

A petition has been set up calling for the 6 main supermarket chains to commit to providing Changing Places toilets in their stores within the next 18 months. Please consider signing this petition.

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Do You Know Someone Who Deserves or Needs a Brand New Garden/Home Makeover? Do You Know Someone Who Deserves or Needs a Brand New Garden/Home Makeover?

22 November 2017 / Posted in: Our Community

ITV1's garden makeover show 'Love Your Garden with Alan Titchmarsh' is back (along with sister show, 'Love Your Home and Garden'). Apply or nominate someone who could benefit.

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