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Latest News: Research

Clinicians' Open Letter about the EAP for Children with SMA Type 1 in the UK

06 April 2017 / Posted in: Research

Today, seventy clinicians, physiotherapists, study coordinators, industry and advocacy groups' representatives, including SMA Support UK, outline their position and recommendations to NICE, NHS England's Care and Clinical Reference Groups (CRGs), NHS Trusts and Biogen.

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AveXis announce results from AVXS-101 Phase I trial

05 April 2017 / Posted in: Research

AveXis has released results from the completed Phase I trial of their gene therapy drug for SMA, AVXS-101.

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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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