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Questions and Answers session with Roche about Risdiplam

  • 21 August 2020

We joined a session with Roche earlier this week to ask them questions following their recent announcement about risdiplam. The following FAQs have been checked for accuracy with Roche where they relate specifically to risdiplam.
Questions and answers apply to people living in England, Scotland, Northern Ireland and Wales.

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What a community! Thank you.

  • 30 October 2020

Today TreatSMA made submissions to NICE to help with the appraisal of Risdiplam to be made available through NHS. We have included extensive data collected from community (over 300 responses in total, so massive thank you).

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EAMS update and how you can help

  • 21 October 2020

You may remember some time ago we posted that MHRA had approved an EAMS process for accessing risdiplam. Just to remind everybody what an EAMS process is, it stands for Early Access to Medicines Scheme, and is designed to provide those who currently have unmet clinical needs to get access to life-saving treatment before it is licensed in the UK.

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Biogen Release a Community Update

  • 6 October 2020

This is a community update from Biogen on the efficacy of Spinraza in patients with SMA Type 2 and 3. Positive improvement were seen in both Types. For more details please see below. Biogen Community Update

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