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Latest News: Research

Waiting for a Place on the UK Nusinersen Expanded Access Programme for SMA Type 1?

04 July 2017 / Posted in: Research

Please help progress access to the programme by letting SMA Support UK know about your child and where you live so that we can discuss the national picture with clinicians and NHS funding bodies.

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Biogen SMA Community Update re: nusinersen

03 July 2017 / Posted in: Research

This shares the information discussed on the SMA Europe call on June 9th – most of which we have already published but it is a useful summary of their position and what they are doing now.

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European Medicines Agency (EMA) report on Spinraza

27 June 2017 / Posted in: Research

You can now read the detailed science behind the European Commission decision to authorise the marketing of nusinersen in Europe. This includes full details of the various clinical trials there have been.

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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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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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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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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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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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European Commission approves nuisnersen to be marketed as Spinraza for those with 5qSMA which includes SMA Types 1, 2 and 3

01 June 2017 / Posted in: Research

We should now hear soon if there will be a Ministerial referral to NICE for appraisal for funding by the NHS.

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