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Latest News: Research

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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Roche drug RG7916 granted Orphan Drug Status in the US

17 January 2017 / Posted in: Research

The US Food and Drug Administration (FDA) has granted Orphan Drug Status to RG7916, a splice-modifying drug developed through a partnership between Roche, PTC Therapeutics, and the SMA Foundation.

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Working to speed up the nusinersen (Spinraza) appraisal by the European Medicines Agency (EMA)

12 January 2017 / Posted in: Research

The first step towards possible licensing of Spinraza in the UK is for it to go through the EMA appraisal process. Read the joint letter sent by UK SMA charities asking the EMA to speed this up. Download a template letter if you want to write to your own MP.

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Novel Antisense Therapy Removes the Brakes on SMN Protein Production

11 January 2017 / Posted in: Research

A potential new therapy for SMA, which could possibly be combined with other drugs such as Spinraza to enhance clinical effectiveness, has been identified and successfully tested in mouse models of the disease.

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