TreatSMA

Survey to explore the experiences of people who are on Risdiplam.

TreatSMA is asking for help from those that are receiving  Risdiplam. We desperately need you to fill in this questionnaire on behalf of yourself or your child to help us in our fight to access Risdiplam here in the UK.

Anyone in any country or on any Risdiplam trial can take part regardless of age or type.

We are looking for at least 50 participants to take part in the survey over the next 72hrs.

Please help us bring this drug to the UK for those still waiting by filling in the survey here.

Thanks,

TreatSMA

Latest Posts

  • Calling on all the community to ACT NOW to avoid losing treatments!

    Calling on all the community to ACT NOW to avoid losing treatments!

    Currently, there are two approved treatments for SMA: risdiplam and nusinersen. These treatments were initially approved temporarily under Managed Access Agreements while the NHS and NICE gathered data to assess their cost-effectiveness. Now, as these agreements come to an end, both treatments face a crucial review.

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  • NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam

    NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam

    SMA UK are proud to join together with Treat SMA and MDUK to bring you this webinar about NICE’s upcoming Multi Technology Assessment for Spinraza and Risdiplam. 2024 sees the end of the managed access agreements (MAAs) for the two treatments. An expert committee will gather to assess the new clinical and real-world evidence, along…

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  • Let the battle begin

    Let the battle begin

    This year the Managed Access Agreement for Spinraza and Risdiplam expires. The MAA was put in place five years ago so the pharmaceutical companies could gather more evidence to resubmit to NICE for approval on the NHS with Biogen extending their MAA by a year.

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