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Latest News: Research

What Happens After a Successful Clinical Trial

06 January 2017 / Posted in: Research

In this timely article, Dr. Alex Murphy describes the organisations involved and outlines the processes that have to be gone through before a drug can be prescribed for an individual in the UK.

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Spinraza (nusinersen) approved by FDA in the US

04 January 2017 / Posted in: Research

Read a joint statement from SMA Support UK, Muscular Dystrophy UK and The SMA Trust.

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Update from Roche on their Clinical Trials for 2017

22 December 2016 / Posted in: Research

Read a statement from Roche about their 2017 clinical trials of drug RG7916.

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Update on Nusinersen Expanded Access Programme in the UK

21 December 2016 / Posted in: Research

Read the report on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1.

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Nusinersen Phase 2 Trial Results Published in The Lancet

15 December 2016 / Posted in: Research

Results from a phase 2 clinical trial of the Ionis Pharmaceuticals gene therapy drug, Nusinersen (now marketed as Spinraza), have been published in the medical journal The Lancet.

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New Clinical Care Research Article: Complementary & Alternative Therapies

06 December 2016 / Posted in: Research

In Dr. Alex Murphy's latest article, he summaries some of the more common CATs (Complementary and Alternative Therapies) and looks into evidence for their use in SMA or similar conditions.

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PhD Studentships at Royal Holloway, University of London

05 December 2016 / Posted in: Research

Applications are open for PhD Studentships and one of the available projects is 'Psychosocial and ethical issues in perinatal gene and cell therapies for Spinal Muscular Atrophy'. Deadline for applications if Friday 9th December.

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Biogen and Ionis Provide Community Update on CHERISH Nusinersen Trial

08 November 2016 / Posted in: Research

Biogen and Ionis Pharmaceuticals have provided the following community statement regarding the interim analysis of the CHERISH trial.

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New Pivotal Study Announced by AveXis

07 November 2016 / Posted in: Research

AveXis has announced details about a future pivotal trial of their gene therapy drug for SMA Type 1, AVXS-101.

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FDA Accept Nusinersen For Priority Review

31 October 2016 / Posted in: Research

Biogen have announced that their New Drug Application (NDA) for Nusinersen has been accepted by the US Food and Drug Administration (FDA) for Priority Review.

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