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Latest News: Research

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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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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