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Latest News: Research

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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Positive News for GOSH children with SMA Type 1

01 June 2017 / Posted in: Research

Families with eligible children with SMA Type 1 currently under Great Ormond St Hospital’s care, who wish to access the nusinersen EAP, will be able to do so over the next 3 months. This includes children under GOSH’s care who currently access treatment in Europe.

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Access to nusinersen in the UK

30 May 2017 / Posted in: Research

We are working with Muscular Dystrophy UK, the SMA Trust and TreatSMA. Read about our focus now and how you can help.

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Cytokinetics Drug Awarded Orphan Drug Designation in the US

25 May 2017 / Posted in: Research

Cytokinetics has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its compound CK-2127107 for the potential treatment of SMA.

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Read the Latest Responses from Biogen about Nusinersen in the UK

16 May 2017 / Posted in: Research

Biogen responds to NHS England's requests about the UK EAP for infants with SMA Type 1; they also tell us when they hope to hear from NICE.

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Biogen Advises on Future Access Position for Children Enrolled in an SMA Type 1 EAP

11 May 2017 / Posted in: Research

We asked about the position in the UK and overseas.

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Update on NHS England's position re EAP for SMA Type 1

10 May 2017 / Posted in: Research

You can now read the latest information about NHS England’s position on considering the possibility of supporting the costs of delivering the EAP in England. We now have authority to share information from the meeting we attended on 25th April with the NHS England Specialised Commissioning team.

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