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AveXis gene therapy trial in UK

  • 10 August 2018

In the next few weeks we are expecting the AveXis AVXS-101 trial to start enrolling. There will be two site centres here in the UK: Great Ormond Street Hospital for Children Contact : [email protected] The John Walton Muscular Dystrophy Research…

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August is SMA Awareness Month

  • 1 August 2018

It's August and its SMA Awareness Month. Please take time to view and share our video. Lets TreatSMA in all its forms, and lets get more treatments as soon as we can to help all.

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A potential side effect added to Spinraza label

  • 30 July 2018

The European label of Spinraza will soon list a new potential side effect: hydrocephalus. We don’t yet know whether this severe condition, which was experienced by only five patients treated with Spinraza, is at all linked to nusinersen, the active ingredient of Spinraza. In response to the Community’s queries Biogen has provided the following update:

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Risdiplam update from Roche

  • 6 July 2018

On 5th July Roche sent us the following information, as per our request to be kept up to date on their current research regarding the RG7916 development programme which will now be known as “risdiplam”. The programme has now reached…

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Spinraza appraisal at NICE

  • 30 June 2018

As everyone knows, TreatSMA along with other patient advocacy groups attended and contributed to an important meeting for Spinraza appraisal at NICE HQ in Manchester on the 27th June.

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