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Nusinersen patient Q&As

  • 19 May 2020

Patient organisations have asked for NICE and NHS to answer a few very important questions with regards to MAA and access to Nusinersen. We are now able to share their answers in the attached documents. We will continue to push for expanded access for this treatment to make sure that everybody gets a chance.

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AveXis gets EC approval for Zolgensma

  • 19 May 2020

AveXis, a Novartis company, announced today that the European Commission (EC) have granted conditional approval for Zolgensma for the treatment of patients with 5q SMA, activating a ‘Day One’ access programme.

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New Trustee announcement

  • 30 April 2020

TreatSMA are extremely happy and proud to announce that Marni Smyth has become the sixth trustee that has joined our team. Marni has been in discussions with TreatSMA for a number of weeks and we take great pleasure in announcing the news.

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Roche reach out to TreatSMA to provide assurances regarding Risdiplam

  • 28 April 2020

Last weekend we announced on our website the changes that Roche were making to their compassion access program in light of Covid-19 challenges. While this was a welcome move we also published a statement questioning the decision not to go further with access. Following that statement we held a call with Roche and their UK Managing Director: We wanted to provide you a summary of that conversation.

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