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Rare Disease UK Announces New Project

26 June 2017 / Posted in: Information & Support

Rare Disease UK will be coordinating a new project that aims to find out more about children and young people’s experiences of living with a rare, genetic or undiagnosed condition.

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The 'Secret Life Of Us' Campaign

23 June 2017 / Posted in: Information & Support

The Disabled Children’s Partnership are launching the 'Secret Life of Us' campaign on 28th June, joining forces to fight for better health and social care for disabled children, young people and their families in England.

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AVXS-101 Gene Therapy Manufacturing Process Streamlined

22 June 2017 / Posted in: Research

AveXis has announced that the process to manufacture their gene therapy drug for SMA, AVXS-101, is now aligned with expectations of the US Food and Drug Administration (FDA).

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How long will the UK EAP for eligible children with SMA Type 1 be open?

16 June 2017 / Posted in: Research

Today, Biogen confirmed that the actual date of closure of the EAP for new children will be agreed on a country by country basis. We are asking for it to stay open as long as possible in the UK and for the earliest possible advice as to when this closure date will be.

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New Clinical Care Research Article by Dr Alex Murphy

15 June 2017 / Posted in: Research

In this article, Dr Alex Murphy discusses the research carried out into three rare sub-types of SMA: Kennedy's, SMARD, Distal.

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Meet the New UK SMA Patient Registry Curator!

15 June 2017 / Posted in: Research

Jo Bullivant has recently taken over as Registry Curator and here she explains a bit more about her role and background.

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Biogen Announces European Ex-factory List Price for Nusinersen

09 June 2017 / Posted in: Research

Expected to be up to €270,000 (approximately £235,000) per individual per year for the three-dose maintenance year but the prices the NHS pays for medicines are not necessarily a reflection of the list prices.

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Quiz Night for SMA Support UK! Quiz Night for SMA Support UK!

09 June 2017 / Posted in: Help Us

Chartered Accountants and Business Advisors, Johnston Smillie, hosted a quiz night last week and raised a fantastic £1,100!

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Can you Help Describe the Experience of Living with SMA?

08 June 2017 / Posted in: Research

Individuals with SMA and caregivers are wanted by Adelphi Values to take part in a one-hour phone interview about their experience of SMA. Participants will receive £50.

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Universal Credit

08 June 2017 / Posted in: Information & Support

Contact a Family launched their Universal Credit #KnowYourClaim campaign on 10th May. This was to make sure people know about Universal Credit and how it might affect families.

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