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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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New SMA Voices: A Dad's Story

04 January 2017 / Posted in: Information & Support

In this new SMA Voices piece, Joseph's dad, Aidan, shares his thoughts, feelings and experiences twenty years on about his son who had SMA Type 1 and sadly passed away.

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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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Supporters of the Month: December 2016 Supporters of the Month: December 2016

15 December 2016 / Posted in: Our Community

This month, we want to say a big thank you to everyone who, this year, has given their time and supported our work in so many different ways.

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Turn2us Launches 'No Cold Homes' Campaign

14 December 2016 / Posted in: Information & Support

Turn2us' poverty campaign, No Cold Homes, is running throughout the Winter and they are promoting the support available for people who can’t afford to heat their homes this Winter.

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Middle Aged Men in Lycra (MAMIL) take on Cycling Challenge! Middle Aged Men in Lycra (MAMIL) take on Cycling Challenge!

07 December 2016 / Posted in: Help Us

Martina and Glyn’s son Max had SMA Type 1 and sadly died in May this year. Glyn and friends decided to form this stylish team to raise money in Max’s memory for Welsh children’s hospice Ty Hafan and SMA Support UK.

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