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Latest News: Research

Government Urged to Re-think its Proposed Changes to the HST Programme

29 March 2017 / Posted in: Research

SMA Support UK will today join Genetic Alliance UK and MDUK at the All Party Parliamentary Group for Muscular Dystrophy which NICE will also attend.

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The D4D Project

28 March 2017 / Posted in: Research

A workshop is taking place in London in January for people with a lived experience of genetic conditions as part of a new research project.

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Nusinersen Expanded Access Programme - What You Can Do To Help

17 March 2017 / Posted in: Research

Our current focus is on trying to get NHS England to agree a potential commissioning framework to support the provision of this new and first treatment for SMA. Find out how you can support these efforts by contacting your local MP with this template letter.

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New Gene Therapy With Potential For SMARD

08 March 2017 / Posted in: Research

New pre-clinical research has shown the exciting potential of using viruses to treat Spinal Muscular Atrophy with Respiratory Distress (SMARD), a recessive motor neuron disease similar to SMA.

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Biogen update on the nusinersen EAP for those with SMA Type 1 in the UK

07 March 2017 / Posted in: Research

In response to our request for an update, Biogen has released this statement today.

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SMA Support UK and the SMA Trust at Rare Disease Day 2017

02 March 2017 / Posted in: Research

The charities shared a stall at Royal Holloway’s annual Rare Disease Day event on February 24th.

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New European Pivotal Study Announced by AveXis

09 February 2017 / Posted in: Research

AveXis has announced details of a European pivotal trial of their gene therapy drug for SMA, AVXS-101.

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Update on the UK Expanded Access Programme (EAP) for Nusinersen for Children with SMA Type 1

20 January 2017 / Posted in: Research

SMA Support UK, Muscular Dystrophy UK and the SMA Trust have been talking with Biogen about what progress there has been on extending the EAP in the UK. You can read the update here.

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Biogen and SMA Europe Provide Community Update on Nusinersen

19 January 2017 / Posted in: Research

In response to a request from SMA Europe, Biogen and the board members of SMA Europe had a call on Wednesday 11th January 2017 to discuss the latest update on nusinersen.

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Joint Charities’ update following the NICE Scoping workshop (13th January 2017)

19 January 2017 / Posted in: Research

Representatives from SMA Support UK, MD UK, The SMA Trust and others attended a workshop which looked at the draft remit and scope for the proposed Technology Appraisal of nusinersen for treating spinal muscular atrophy.

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